Category Archives: Genetic Therapy

Sanofi is handing back rights to two gene therapy programmes it acquired from Oxford Biomedica more than 10 years ago.

In 2009 the French drug giant licensed Oxford Biomedica's SAR422459 for Stargardt disease, a genetic eye disorder that causes progressive degeneration of the macula, and SAR421869 for Ushers Syndrome type 1b, a recessive genetic disease causing deafness from birth and subsequent progressive vision loss.

However, following completion of a portfolio review, and an earlier announcement that it was seeking partners for these programmes, Sanofi is now gearing up to hand back rights to them to Oxford Biomedica.

The timing of return of these programmes and operational details are yet to be determined. However, when the rights to the two programmes are returned, the group will undertake its own internal evaluation to determine the potential future for these programmes and decide whether to commit further resources to them, the UK biotech said in a statement.

Follow this link:
Sanofi hands back rights to Oxford Biomedica gene therapies - PharmaTimes

People who survive childhood cancers are at higher risk for getting cancer again later in life.

Some of the risk is a side effect of treatment, as both chemotherapy and radiation cause genetic damage that can lead to future cancers.

But risk of second cancers is highest among those who require intensive treatment and also have genetic mutations that make them less able to repair damaged DNA, researchers say.

Damage from chemotherapy and radiation treatment can cause unrelated cancers as well.

However, researchers from St. Jude Childrens Hospital in Tennessee say that the side effects of treatment only explain part of the risk of secondary cancers.

We identified mutations affecting specific types of DNA-repair mechanisms, which combined with certain intensities of therapies, could dramatically increase the risk of developing subsequent cancers like breast cancer, sarcoma, and thyroid cancer, said Na Qin, PhD, of St. Judes department of epidemiology and cancer control, and one of the co-authors of a new study published in the Journal of Clinical Oncology.

Several studies have investigated the long-term effects of cancer on childhood survivors, but weve never had data that explained why specific children are at higher risk for subsequent cancer, Sean Marchese, a registered nurse with The Mesothelioma Center and a medical oncology expert, told Healthline.

With these findings, physicians can identify which cancer treatments to avoid and prevent an increased risk of adult cancer for children with specific genetic mutations. Gene therapy is already informing us which medicines are useful in particular patient populations.

Now gene sequencing may let us know when and how to protect high-risk childhood cancer survivors from subsequent cancer recurrences, he said.

In the new study, researchers looked at blood samples from 4,402 pediatric cancer survivors collected through the ongoing St. Jude Lifetime Cohort.

The researchers evaluated 127 genes from six genetic pathways involved in repairing DNA.

The data collected from the samples was then cross-referenced with the cumulative doses of chemotherapy and maximum dose of region-specific radiotherapy given to children during their initial cancer treatment.

Zhaoming Wang, PhD, a corresponding study author from St. Judes departments of epidemiology and cancer control and computational biology, told Healthline that individuals who had high exposure to chemotherapy and radiation as well as inherited mutations that made their DNA-repair genes less effective were found to be most at risk for future cancers.

Wang explains that mutated DNA-repair genes are less able to fix genetic damage caused by cancer therapy, thus raising the risk of future cancers.

We already knew that the larger the dose, the more DNA damage, he said. Now we know that genetics play a role in secondary cancer risk.

Those with low exposure to gene-damaging therapy and one or more of 538 germline mutations in 98 DNA-repair genes studied were at the next highest risk level.

They were followed by those who didnt undergo chemo or radiation but had mutations.

The people with the least risk of occurring cancers were those who had no exposure to chemotherapy or radiation treatment and had no genetic mutations, researchers reported.

The different risk profiles were stark.

For individuals over age 45 with a history of breast cancer, for example, the risk of second cancer was 49 percent among those who had experienced intensive therapy and also had genetic mutations.

The risk dropped to 14 percent among those who had received low chemotherapy or radiation and had mutations.

It was 10 percent among those who received no therapy but had mutations.

Among breast cancer patients with no therapy and no mutations, the risk of a recurring cancer was just 2.5 percent.

We know that people with a high dose of radiotherapy or chemotherapy are at high risk, and this group of people could be prioritized for genetic screening that would refine their risk profile even further, explained Wang.

He notes that a targeted genetic test could be conducted on high-risk patients for less than $1,000.

Of the 4,402 pediatric cancer survivors, 495 developed 1,269 secondary cancers.

The latest study builds upon previous St. Jude research on the risk of recurring cancers among pediatric breast cancer patients.

View original post here:
Children Who Survive Cancer Higher Risk of Secondary Cancer as Adults - Healthline

Several projects that push the boundaries of knowledge and have high potential for impact have been awarded support through Princeton'sDean for Research Innovation Fund.

The funding makes possible explorations in the natural sciences and social sciences, collaborations with industry, and collaborations between artists and scientists or engineers. Several of the projects have the potential for direct benefits to human health while others explore themes in history and the arts. The projects were chosen by faculty-led committees based on the quality, originality and potential of the research.

"Princeton faculty are pioneers across the range of human inquiry, and these innovation funds enable our researchers and their teams to explore paths that they might not otherwise take," said Dean for ResearchPablo Debenedetti, the Class of 1950 Professor in Engineering and Applied Science and professor of chemical and biological engineering. "Through this funding program, the University shows support for the innovation mindset that leads to truly profound advances and can also lead to societal benefits."

A project to study antimicrobial agents found in the pouches of marsupials such as this sugar glider is one of three to be awarded Dean for Research Innovation Funds for New Ideas in the Natural Sciences.

Photo by Patrick Kavanagh

The fund supports the exploration of new concepts that require additional study or experiments before they are ready to become the basis of a competitive proposal to a funding agency.

Ricardo Mallarino, assistant professor of molecular biology, will study the naturally occurring antimicrobial agents that marsupials which carry their offspring to term in an external pouch employ to kill toxic microbes. Marsupials are born without a working immune system and rely for survival entirely on factors secreted by glands found in the pouch or transferred through maternal milk. The researchers will explore the genetic sequences of these molecules, known as antimicrobial peptides, to discover how the genes have evolved over time in ways that contribute to their efficacy against microbes, with the eventual goal of understanding natural defense mechanisms and informing the design of new antimicrobial drugs.

Charles Fefferman and Amit Singer

Photos by Julia Fefferman and provided by the Department of Mathematics

Amit Singer, professor of mathematics and the Program in Applied and Computational Mathematics, and Charles Fefferman, the Herbert E. Jones, Jr. '43 University Professor of Mathematics, will develop algorithms to enhance the capability of a powerful microscope to capture images of protein structures as they move and change shape. The team will adapt mathematical approaches for use with cryogenic electron microscopy (cryo-EM) to take pictures of not just fixed molecules but also ones that are flexing and morphing into differing shapes, as happens for example when a drug molecule interacts with its target receptor.

Lisa Boulanger, associate professor of neuroscience, will conduct research to improve the safety and efficacy of viruses that are used for gene therapy to treat nervous system disorders including spinal motor atrophy and certain kinds of blindness. The most commonly used viruses are forms of adeno-associated virus, or AAV, in which the viral genes were removed and replaced with therapeutic genes. Boulanger found that these viruses can unexpectedly change the structure of circuits in the brain, changes that are associated with increased exploratory behavior in mice. She and her team will determine how AAV alters neural circuits, screen for approaches to prevent these changes, and facilitate gene therapy in the nervous system.

The use of individual-level historical data for studies of long-term impacts of history, including precolonial and colonial societies in Africa, is the focus of a new projected awarded the Dean for Research Innovation Fund for New Ideas in the Social Sciences.

Image by Franois-Edmond Fortier

This fund encourages scholarship on new and enduring questions. The selected projects are ones that will result in the advancement of a discipline through the development of new directions, working groups, conferences, technologies, or expanded access to research resources, or lead to a major piece of scholarly work.

Katja Guenther, associate professor of history, will explore the concept of resilience, which has emerged over the last few decades as one of the most prized traits of the modern age, the key to the future success of our businesses, financial systems, cities, the environment, and even humanity. Guenther will trace the history of the concept across a range of disciplines ranging from psychology to ecology while incorporating environmental humanities, the history of science and the study of systems. In examining the way resilience has been used in the past, she will trace its ethical ramifications, its translations into policy, and its future implications.

Leonard Wantchekon,professor ofpolitics andinternationalaffairs, will lead a team of undergraduate and graduate studentsfrom Princeton University as well as researchers from the African School of Economics and the Universidad de los Andesto explore how to rigorously collect individual-level historical data for studies of long-term impacts of historicalevents.Through studies of precolonial and colonial societies in Africa and the Americas, Wantchekon will develop a novel approach that combines econometrics and advanced statistical methods with more traditional historical and social research techniques to pioneer the emerging field of historical applied microeconometrics.

This fund supports research collaborations that address societal challenges of interest both to industry and to academic scientists and engineers. Industry often plays an essential role in bringing the innovations of University researchers to fruition and making them available to society at large. The program requires an agreement from the industry collaborator to provide matching funds in the second year of the project.

Gerard Wysocki, associate professor of electrical engineering, and his team will develop a highly sensitive laser-based sensor in collaboration with Hamamatsu Photonics, a leading instrumentation company. During critical care of patients in respiratory distress, measuring the consumption of oxygen in the breath can provide important information about the patient's health. The resulting prototype, capable of detecting not only oxygen concentration but the types (or isotopes) of oxygen, will be evaluated by collaborators in a hospital setting.

Howard Stone, Princeton's Donald R. Dixon '69 and Elizabeth W. Dixon Professor of Mechanical and Aerospace Engineering, will team with Princeton, NJ, medical device firm NovaFlux Inc. to explore the ability of a new cleaning fluid composed of nano-sized fibers and micro-sized particles suspended in a liquid. Stone and colleagues at NovaFlux will explore the fluid's ability to remove bacterial coatings, or biofilms, which form on surfaces such as medical tubing and devices. The fluid can also be used to remove a broad range of pathogens including bacteria, spores and viruses.

Left: Dancer Cori Kresge in photo by Sigrid Adriaenssens. Right: Cori Kresge in net, with dancer Chris Ralph standing in background.

Photo by Barry Onouye Studio

This fund encourages collaborations between faculty scholars in the arts and those in the natural sciences or engineering to promote synergistic innovations, allowing experts in seemingly unrelated fields to unify and expand their respective knowledge in ways that benefit both disciplines.

Sigrid Adriaenssens and Rebecca Lazier

Photos by Sameer A. Khan/Fotobuddy and Bentley Drezner

Combining dance and structural engineering, Rebecca Lazier, senior lecturer in dance in the Lewis Center for the Arts, and Sigrid Adriaenssens, associate professor of civil and environmental engineering, will collaborate on a new project involving the movements of dancers as a strategy to explore nets as resilient building facades and impact barriers. Nets are attractive for uses that require flexibility to withstand waves, winds and earthquakes, but they stiffen with usage, making for potentially disastrous outcomes. This project aims to create choreographic works that generate a new understanding of how nets turn rigid when loaded and soften when unloaded. At the same time, dancers will explore the interaction between net and human. The net will be a full collaborative partner in the dance rather than a setting or a prop, enabling creativity that moves beyond how people have used nets previously.

The project involves first designing and fabricating different types of nets and then hosting intensive research sessions with dancers in Princeton's Lewis Center for the Arts. The team will experiment with choreography and collect movement in a digital framework gathered from reflective beads mounted on the nets. The project will include collaboration with Adam Finkelstein, professor of computer science, to transform these data into insights on net structure and properties. The project will include a public performance.

Clockwise from top left: Eduardo Cadava, Fazal Sheikh, Mark Zondlo and John Higgins.

Photos by Eduardo Cadava, Alexandra Beck, Andlinger Center for Energy and the Environment, and Laura Pedrick

A team of artists, scientists and engineers led by Eduardo Cadava, professor of English,John Higgins, associate professor of geosciences, and Mark Zondlo, associate professor of civil and environmental engineering, will embark on a field measurement and outreach project centered on environmental justice issues in the Red Rock Wilderness surrounding Bears Ears and Grand Staircase-Escalante National Monuments in southern Utah. The project, which builds on work by artist and photographer Fazal Sheikh, the 2018-19 Barron Visiting Professor in the Environment and the Humanities and current artist-in-residence at the Princeton Environmental Institute (PEI), will document the effects of extractive industries such as mining on public and Native lands. Expected outcomes include collaborations with indigenous communities and native engineers and scientists, methane and water testing, geological research, an interactive mapping system and website, and online syllabi. The project will contribute to an exhibition at the Smart Art Museum and the Yale University Gallery of Art.

The team includes Michael Celia, the Theodora Shelton Pitney Professor of Environmental Studies and professor of civil and environmental engineering and director of PEI; Peter Jaffe, the William L. Knapp '47 Professor of Civil Engineering and professor of civil and environmental engineering; Zia Mian, research scientist at Princeton's Woodrow Wilson School of Public and International Affairs and co-director of the Program on Science and Global Security; and Emily Wild, Princeton's chemistry, geosciences and environmental studies librarian.

Continue reading here:
Funding the next big idea: New projects receive Dean for Research Innovation awards - Princeton University

Wed, Jun 10, 2020 - 5:50 AM

THE world is engaged in a war against Covid-19 - and the health sector is on the front line. The good news is that the race to develop treatments for Covid-19 has been remarkable for both its speed and scale, according to the Vaccine Alliance, a global health partnership.

So far, companies, scientific groups and institutions around the world have announced efforts to address Covid-19.

Signs of resilience

Healthcare has both been one of the strongest performing sectors over the past few years, and one of the most resilient so far during the Covid-19 crisis.

That healthcare has been relatively resilient is, to an extent, to be expected: After all, individuals still need to seek medical care, even in a recession and health needs become amplified during these times.

In fact, the US healthcare sector has outperformed the market in recent months but also over a longer period.

For example, the S&P US healthcare returned 4.3 and 34.9 per cent in the past six months and three years, respectively, while the general market was down 3.1 and up 25.3 per cent respectively.

Bifurcation of performance

But healthcare faces some headwinds too - meaning there could be some short-term volatility for the sector. Social distancing is having significant economic ramifications, and healthcare companies have not been completely spared. Healthcare providers have been the hardest hit in the sector as almost all elective activity has been suspended to focus on containing and treating Covid-19

The pandemic's effect on healthcare stocks through the first quarter of 2020 has been varied. Performance within biopharmaceuticals was bifurcated. Companies expected to play a role in either developing or partnering to deliver a Covid-19 therapy or vaccine outperformed significantly.

At the same time, many innovative small-cap drug stocks underperformed as a result of the uncertainty surrounding how the current health crisis might impact their ability to start or complete important clinical trials.

While a number of companies are working on antiviral and vaccine candidates for Covid-19, a vaccine may not be available in the US this year. In addition, the economic benefits from any such therapies remain unclear, particularly given the uncertainty of both the duration of the pandemic and a potential vaccine's allowable payment terms.

One approach will be to look at companies that, along with their potential to contribute solutions to the fight against Covid-19, have wider portfolios of attractive therapies and assets.

When the dust settles, many countries will be investing and building up more buffers and resilience in their healthcare systems, to prepare them for possible future pandemics. This could include a rethink in the proximity and flexibility of supply chains and production to be closer to the end-consumer.

Healthcare fundamentals

Notwithstanding challenges like these, the long-term fundamentals supporting the sector remain intact.

First, the world's population is ageing, especially in many developed countries. This is likely to drive demand for healthcare, and boost revenues for the entire industry.

Furthermore, in many developing economies, including China and other parts of Asia, rising wealth and a willingness to spend more on healthcare and quality of life will continue to be a positive for the industry.

Of course, the competitive landscape will also evolve, and not all companies may benefit equally from this - or even benefit at all, for that matter. But at least the overall revenue pie for the industry is likely to grow.

What is more, there is tremendous progress being made in certain segments of the healthcare industry in areas such as the development of medical devices using advanced technology, as well as fields like gene-editing, gene-therapy and precision medicine.

These are all structural developments which we believe are here to stay, and represent potential opportunities for companies and investors.

Investment themes

Clearly, the Covid-19 outbreak removed the behavioural barriers to widespread adoption of telemedicine, ensuring that telemedicine solutions are here to stay.

Genetic therapies could significantly enhance healthcare in a post Covid-19 world. Unlike traditional drugs, genetic therapies aim to cure diseases by modifying or removing faulty human genetic information, actually removing the cause of illness. This represents a paradigm shift in medical care treatments.

How should we invest in the healthcare sector?

Healthcare investing is complex and there is a need to understand the evolving investment themes.

One recommended approach is through active management which offers the benefits of diversification in tandem with skilled stock selection. A typical healthcare fund management team would comprise a large number of specialists who have considerable experience in the entire spectrum of healthcare: biopharma, healthcare services and medical technology. This would give investors an edge to better capitalise on the complex nature of the sector.

Investors can also consider healthcare exchange traded funds (ETFs). ETFs replicate the holdings of healthcare indexes, but are not actively managed. The liquidity of ETFs gives investors the ability to express short term views very quickly, as investors are able to jump into or out of positions frequently. Short-term traders, are able to very quickly make a play on the price swings in the sector with low transaction fees.

More here:
Healthcare sector investing is on the cusp of change - Business Times

Global Gene Therapy for Inherited Genetic Disorders Market analysis 2015-2027, is a research report that has been compiled by studying and understanding all the factors that impact the market in a positive as well as negative manner. Some of the prime factors taken into consideration are: various rudiments driving the market, future opportunities, restraints, regional analysis, various types & applications, Covid-19 impact analysis and key market players of the Gene Therapy for Inherited Genetic Disorders market. nicolas.shaw@cognitivemarketresearch.com or call us on +1-312-376-8303.

Download Report from: https://www.cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-inherited-genetic-disorders-market-report

Global Gene Therapy for Inherited Genetic Disorders Market: Product analysis: Eye Disorders, Hematological Disorders, Central Nervous System Disorders, Muscular Disorders, Others

Global Gene Therapy for Inherited Genetic Disorders Market: Application analysis: Hospital, Clinic, Research Institute, Others

Major Market Players with an in-depth analysis: BioMarin Pharmaceutical Inc., bluebird bio Inc., Novartis AG, Orchard Therapeutics Plc, Spark Therapeutics Inc.

The research is presented in such a way that it consists of all the graphical representations, pie charts and various other diagrammatic representations of all the factors that are used for the research. Gene Therapy for Inherited Genetic Disorders market research report also provides information on how the industry is anticipated to provide a highly competitive analysis globally, revenues generated by the industry and increased competitiveness and expansions among various market players/companies.

Get A Free Sample of Gene Therapy for Inherited Genetic Disorders Market Report: https://www.cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-inherited-genetic-disorders-market-report#download_report

The Gene Therapy for Inherited Genetic Disorders industry is projected in assembling information regarding dynamic approximations and also listings of a profitable progression rate annually in the expected duration according to a recent & latest study. The latest Coronavirus pandemic impact along with graphical presentations and recovery analysis is included in the Gene Therapy for Inherited Genetic Disorders research report. The research report also consists of all the latest innovations, technologies and systems implemented in the Gene Therapy for Inherited Genetic Disorders industries.

Various factors with all the necessary limitations, expenditure/cost figures, consumer behaviour, supply chain, government policies and all the information related to the market have been included in the Gene Therapy for Inherited Genetic Disorders Market report. The research report also provides light on various companies & their competitors, market size & share, revenue, forecast analysis and all the information regarding the Gene Therapy for Inherited Genetic Disorders Market.

Checkout Inquiry for Buying or Customization of Report: https://www.cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-inherited-genetic-disorders-market-report#download_report.

Gene Therapy for Inherited Genetic Disorders Market research report provides an in-depth analysis of the entire market scenario starting from the basics which is the market introduction till the industry functioning and its position in the market as well as all the projects and latest introductions & implementations of various products. The research study has been assembled by understanding and combining various analysis of regions globally & companies and all necessary graphs and tables that bring the theory into an exact representation through numerical values and standard tables.

The global estimations of the market value, market information/definition, classifications of all the types & applications, overall threats & dips that can be assumed and many other factors which consist the overall market scenario and its happening globally along with the forthcoming years are compiled in the Gene Therapy for Inherited Genetic Disorders market research report. Hence this report can serve as a handbook/model for the enterprises/players interested in the Gene Therapy for Inherited Genetic Disorders Market as it consists all the information regarding the Gene Therapy for Inherited Genetic Disorders market.

Any query? Enquire Here For Discount (COVID-19 Impact Analysis Updated Sample): Click Here>Download Sample Report of Gene Therapy for Inherited Genetic Disorders Market Report 2020 (Coronavirus Impact Analysis on Gene Therapy for Inherited Genetic Disorders Market)

Note In order to provide more accurate market forecast, all our reports will be updated before delivery by considering the impact of COVID-19.(*If you have any special requirements, please let us know and we will offer you the report as you want.)

About Us: Cognitive Market Research is one of the finest and most efficient Market Research and Consulting firm. The company strives to provide research studies which include syndicate research, customized research, round the clock assistance service, monthly subscription services, and consulting services to our clients. We focus on making sure that based on our reports, our clients are enabled to make most vital business decisions in easiest and yet effective way. Hence, we are committed to delivering them outcomes from market intelligence studies which are based on relevant and fact-based research across the global market.Contact Us: +1-312-376-8303Email: nicolas.shaw@cognitivemarketresearch.comWeb: https://www.cognitivemarketresearch.com/

**********Download the Entire Report*************************************************https://www.cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-inherited-genetic-disorders-market-report

More here:
Global Gene Therapy for Inherited Genetic Disorders Market will Witness Steady Growth Till 2027 Post COVID 19 Pandemic, Top Manufactures BioMarin...

Current treatments for hearing loss focus on amplifying sound, rather than targeting the cause of the disease. Now, a team of researchers report the use of base editors to treat Baringo micea mouse model of recessive genetic deafness caused by a point mutation in the transmembrane channel-like 1 gene Tmc1. This method, which is the first example of repairing a recessive gene mutation, restored hair cell sensory transduction and partial hearing.

David Liu, PhD [Casey Atkins]Details about the approach are published in the paper, In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness, in Science Translational Medicine.

Base editing is a technique developed by the lab of David Liu, PhD, director of the Merkin Institute of Transformative Technologies in Healthcare and professor of chemistry and chemical biology at Harvard University, that can specifically correct a common class of base substitutions without cleaving DNA. Base editing has the potential to provide restoration of gene function by directly repairing point mutations.

This research is very important for the pediatric community here at Boston Childrens Hospital and elsewhere because about 4,000 babies are born each year with genetic hearing loss, said Jeffrey Holt, PhD, director of otolaryngology research at Boston Childrens Hospital and co-senior author on the paper. And, we feel this is a big step beyond the field of hearing restoration and for the broader field focused on the treatment of genetic disorders.

The gene editing method included a cytosine base editor inserted into a dualadeno-associated virus (AAV) system, which was injected into the inner ear of the animals with hearing loss. The treatment reversed the mutation, improved sensory transduction, and partially restored hearing in mice, following a single injection of base editor AAVs. This technique opens the door to treating other genetic forms of hearing loss and other genetic diseases

Earlier research in 2015 from the Holt lab and colleagues showed that replacing a full DNA sequence for Tmc1 into the sensory cells in the ear restores hearing in deaf mice. The Tmc1 protein is required for normal auditory function because it forms mechanosensitive ion channels in sensory hair cells of the inner ear.

In that case, we used a single engineering AAV to deliver a functioning copy of the Tmc1 gene into the ear, Holt said.

This research goes a step further. Instead of replacing a gene, the team repaired a single mutation in the Tmc1 gene converting it back to the correct sequence. Its like your spell-checker, Holt explained. If you type the wrong letter, spell checker fixes it for you. When the team fixed the defect in the sensory cells in the ear, the edited cells recovered 100% of their function.

One limitation of AAV vectors is their limited capacity. In this case, the base editor was too large for a single AAV. As a result, the base editor sequence was split into two AAVs. The technique required the use of two viral vectors to deliver the base editing machinery. Cells with both vectors recovered 100% function.

Once the cell was infected with these two parts, it was able to reassemble into a single full length sequence and then perform the base editing task we needed, said co-first author Olga Shubina-Oleinik, PhD, a post-doctoral fellow at the Holt lab.

Although the approach worked when both AAVs made their way into the cell, that was the case in about one-quarter of the cells, which was enough to provide some hearing to the mice.

We got it to work but we need to boost the efficiency to make it broadly useful, said Holt. If only one AAV got into the cell, it did not work. But the message is that when we got both into the cells, we went from zero function to 100%. That tells me all we need to do is get it into more cells and we will recover more hearing function.

At least 100 different genes are involved in hearing in the inner ear. Mutations in any one of those can lead to hearing loss.

We have been developing different strategies targeting several of these different forms of hearing loss, said Holt. It really takes a precision medicine approach where we are trying to tailor our strategy specific, not just each gene that is involved, but in some cases the individual genetic mutation in the gene as is the case with this study.

The Holt lab has a long history of success unraveling these genetic causes of hearing loss and developing gene therapy treatments for genetic forms of hearing loss. In 2011, the team first discovered that the Tmc1 protein is required for hearing and balance. After its 2015 success, the Holt team used CRISPR-Cas9 gene editing in 2019 to prevent hearing loss in Beethoven mice, a model of a dominant Tmc1 mutation.

Over 70 different mutations have been identified in the Tmc1 gene in humans. We hope this new technique will allow us to pick them off one at a time to restore hearing and balance related to the inner ear, said Holt.

See the original post here:
Base Editing Gene Therapy Restores Hearing in Mice - Genetic Engineering & Biotechnology News