Category Archives: Genetic Therapy

IBNLive.com

PM dishonest, failed to act on time: Yashwant Sinha on economy IBNLive.com Senior BJP leader and former finance minister Yashwant Sinha has hit out at Prime Minister Manmohan Singh accusing him of being "dishonest" by trying to distance himself from the actions taken by the Finance Ministers in his Cabinet. Time dubs Manmohan as 'underachiever'Hindustan Times Congress counters Time magazine's 'underachiever' remark against PMNDTV PM has sent a message of disillusionment, despair: BJPEconomic Times The Hindu -City Journal.in all 7 news articles »

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NEW YORK, July 2, 2012 /PRNewswire/ --Reportlinker.com announces that a new market research report is available in its catalogue:

http://www.reportlinker.com/p0922674/Gene-Therapy-Market-to-2018---Product-Development-Slowed-by-Clinical-Failures-Close-Regulatory-Surveillance-and-High-Compliance-Standards.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Biological_Therapy

Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards

This report is built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis by GBI Research's team of industry experts.

Gene therapies are biological compounds, which modify or replace disease-causing genes. These therapies are the new therapeutic class aimed at treating diseases associated with genetic mutations. Gene therapy promises to provide new treatments for a large number of inherited and acquired diseases. The basic concept of gene therapy is to introduce a piece of genetic material into target cells, which will result in either a cure for the disease or a slowdown in the progression of the disease. It involves the transfer of a functional gene copy into specific cells of an individual in order to repair a faulty gene copy. It may be used to replace a defective gene, or to introduce a new gene to cure a condition.

For example, mutations in genes on the X chromosome lead to X chromosome-linked genetic diseases such as Duchenne muscular dystrophy and hemophilia. Since males have only one copy of the genes from this chromosome, there is no other normal copy available to fulfill a defective gene's function which is present on the X chromosome. If the normal copy of the mutated gene is delivered in the nucleus externally through a delivery agent, the cells can produce the normal gene products and the disease would be treated.

From a commercial perspective, there is a huge unmet need in oncology and autoimmune diseases, amongst others, that could further drive growth of the pharmaceutical and biotech industry. The unmet need is largely driven by the lack of efficacious and safe therapeutic products based on conventional pharmaceutical and biotech research. Gene therapy is a new therapeutic category that has the potential to satisfy this unmet need, especially considering how efficacious and safe this therapeutic category is expected to be.

GBI Research's analysis suggests that therapies developed using gene therapy technology can address the majority of the unmet needs prevailing in the current pharmaceutical market. The inherent structure of gene therapies and their potential to replace the functions of defective genes make them highly effective to knockdown any gene that was previously unapproachable by conventional therapies. Gene therapies are poised to become the next most promising class of drugs in the pharmaceutical industry. Currently there are only three approved products, namely Gendicine, Oncorine and Rexin-G, with a collective market little above $2.8m. Since first movers always have the competitive edge, many large pharmaceutical and biotechnology companies have already commenced their R&D activities on gene therapies.

This report provides insights into the major unmet needs prevailing in the current pharmaceutical industry, and points to gene therapies as the solution to these unmet needs. The report also elucidates the promising late-stage gene therapy pipeline, and provides insights into the gene therapeutics R&D pipeline and funding opportunities.

- Analysis of the leading therapeutic segments for which clinical development in gene therapy is being conducted.

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Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High ...

Hindustan Times

I do not believe in miracles: Pranab jibe at Sangma Hindustan Times "I do not believe in miracles," UPA Presidential nominee Pranab Mukherjee remarked today, taking a jibe at his rival PA Sangma who is hoping for a miracle to see him through in the July 19 poll. "I do not believe in miracles. Talent in Congress, crisis-management not a problem: Pranab MukherjeeIBNLive.com Prez poll: Pranab seeks support of TMC, others who are still undecidedIndian Express all 717 news articles »

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IBNLive.com

Violence in Barak Valley over assault on MLA Rumi Nath Rediff The assault on Congress MLA Rumi Nath sparked off violent protests and a bandh in south Assam's Barak Valley on Sunday as police resorted to lathicharge and apprehended five persons involved in the attack. Tension prevailed for the second day in Barak ... Mob beats Indian MLA for second marriageThe News International Assam: Mob tried to rape me, says Congress MLA Rumi NathIBNLive.com all 113 news articles »

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Moneycontrol.com

BJP top brass confident K'taka crisis will be overcome Zee News New Delhi: BJP top brass is confident of tiding over the present crisis in Karnataka till the presidential poll gets over but appears to be getting increasingly reconciled to replacing Sadanand Gowda with Jagdish Shettar as chief minister soon ... BSY camp sets July 5 deadline to remove GowdaIBNLive.com Will be happy if K'taka assembly polls are held in Dec: EshwarappaDaily News & Analysis Leadership tussle overshadows 4-year-rule of BJP in KarnatakaBusiness Standard Deccan Herald -Sahara Samay all 576 news articles »

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Newswise Researchers at the Buck Institute have corrected the genetic mutation responsible for Huntingtons Disease (HD) using a human induced pluripotent stem cell (iPSC) that came from a patient suffering from the incurable, inherited neurodegenerative disorder. Scientists took the diseased iPSCs, made the genetic correction, generated neural stem cells and then transplanted the mutation-free cells into a mouse model of HD where they are generating normal neurons in the area of the brain affected by HD. Results of the research are published in the June 28, 2012 online edition of the journal Cell Stem Cell.

iPSCs are reverse-engineered from human cells such as skin, back to a state where they can be coaxed into becoming any type of cell. They can be used to model numerous human diseases and may also serve as sources of transplantable cells that can be used in novel cell therapies. In the latter case, the patient provides a sample of his or her own skin to the laboratory. We believe the ability to make patient-specific, genetically corrected iPSCs from HD patients is a critical step for the eventual use of these cells in cell replacement therapy, said Buck faculty Lisa Ellerby, PhD, lead author of the study. The genetic correction reversed the signs of disease in these cells the neural stem cells were no longer susceptible to cell death and the function of their mitochondria was normal. Ellerby said the corrected cells could populate the area of the mouse brain affected in HD, therefore, the next stage of research involves transplantation of corrected cells to see if the HD-afflicted mice show improved function. Ellerby said these studies are important as now we can deliver patient-specific cells for cell therapy, that no longer have the disease causing mutation.

Huntington's disease (HD) is a devastating, neurodegenerative genetic disorder that affects muscle coordination and leads to cognitive decline and psychiatric problems. It typically becomes noticeable in mid-adult life, with symptoms beginning between 35 and 44 years of age. Life expectancy following onset of visual symptoms is about 20 years. The worldwide prevalence of HD is 5-10 cases per 100,000 persons. More than a quarter of a million Americans have HD or are "at risk" of inheriting the disease from an affected parent. Key to the disease process is the formation of specific protein aggregates (essentially abnormal clumps) inside some neurons.

All humans have two copies of the Huntingtin gene (HTT), which codes for the protein Huntingtin (Htt). Part of this gene is a repeated section called a trinucleotide repeat, which varies in length between individuals and may change between generations. When the length of this repeated section reaches a certain threshold, it produces an altered form of the protein, called mutant Huntingtin protein (mHtt). Scientists in the Ellerby lab corrected the mutation by replacing the expanded trinucleotide repeat with a normal repeat using homologous recombination. Homologous recombination is a type of genetic recombination where two molecules of DNA are exchanged. In this case the diseased DNA sequence is exchanged for the normal DNA sequence.

Contributors to the work: Mahru An and Ningzhe Zhang are shared first authors of this study. Other Buck Institute researchers involved in the study include Gary Scott, Daniel Montoro, Tobias Wittkop, and faculty members Sean Mooney and Simon Melov. The work was funded by the Buck Institute and the National Institutes of Health.

About the Buck Institute for Research on Aging The Buck Institute is the U.S.s first and foremost independent research organization devoted to Geroscience focused on the connection between normal aging and chronic disease. Based in Novato, CA, The Buck is dedicated to extending Healthspan, the healthy years of human life and does so utilizing a unique interdisciplinary approach involving laboratories studying the mechanisms of aging and those focused on specific diseases. Buck scientists strive to discover new ways of detecting, preventing and treating age-related diseases such as Alzheimers and Parkinsons, cancer, cardiovascular disease, macular degeneration, diabetes and stroke. In their collaborative research, they are supported by the most recent developments in genomics, proteomics and bioinformatics. For more information: http://www.thebuck.org.

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Scientists Correct Huntington's Mutation in Induced Pluripotent Stem Cells