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What are genome editing and CRISPR-Cas9? – Genetics Home …

Posted: February 16, 2019 at 10:43 am

Genome editing (also called gene editing) is a group of technologies that give scientists the ability to change an organism's DNA. These technologies allow genetic material to be added, removed, or altered at particular locations in the genome. Several approaches to genome editing have been developed. A recent one is known as CRISPR-Cas9, which is short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9. The CRISPR-Cas9 system has generated a lot of excitement in the scientific community because it is faster, cheaper, more accurate, and more efficient than other existing genome editing methods.

CRISPR-Cas9 was adapted from a naturally occurring genome editing system in bacteria. The bacteria capture snippets of DNA from invading viruses and use them to create DNA segments known as CRISPR arrays. The CRISPR arrays allow the bacteria to "remember" the viruses (or closely related ones). If the viruses attack again, the bacteria produce RNA segments from the CRISPR arrays to target the viruses' DNA. The bacteria then use Cas9 or a similar enzyme to cut the DNA apart, which disables the virus.

The CRISPR-Cas9 system works similarly in the lab. Researchers create a small piece of RNA with a short"guide" sequence that attaches (binds) to a specific target sequence of DNA in a genome. The RNA also binds to the Cas9 enzyme. As in bacteria, the modified RNA is used to recognize the DNA sequence, and the Cas9 enzyme cuts the DNA at the targeted location. Although Cas9 is the enzyme that is used most often, other enzymes (for example Cpf1) can also be used. Once the DNA is cut, researchers use the cell's own DNA repair machinery to add or delete pieces of genetic material, or to make changes to the DNA by replacing an existing segment with a customized DNA sequence.

Genome editing is of great interest in the prevention and treatment of human diseases. Currently, most research on genome editing is done to understand diseases using cells and animal models. Scientists are still working to determine whether this approach is safe and effective for use in people. It is being explored in research on a wide variety of diseases, including single-gene disorders such as cystic fibrosis, hemophilia, and sickle cell disease. It also holds promise for the treatment and prevention of more complex diseases, such as cancer, heart disease, mental illness, and human immunodeficiency virus (HIV) infection.

Ethical concerns arise when genome editing, using technologies such as CRISPR-Cas9, is used to alter human genomes. Most of the changes introduced with genome editing are limited to somatic cells, which are cells other than egg and sperm cells. These changes affect only certain tissues and are not passed from one generation to the next. However, changes made to genes in egg or sperm cells (germline cells) or in the genes of an embryo could be passed to future generations. Germline cell and embryo genome editing bring up a number of ethical challenges, including whether it would be permissible to use this technology to enhance normal human traits (such as height or intelligence). Based on concerns about ethics and safety, germline cell and embryo genome editing are currently illegal in many countries.

Gupta RM, Musunuru K. Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9. J Clin Invest. 2014 Oct;124(10):4154-61. doi: 10.1172/JCI72992. Epub 2014 Oct 1. Review. PubMed: 25271723. Free full-text available from PubMed Central: PMC4191047.

Hsu PD, Lander ES, Zhang F. Development and applications of CRISPR-Cas9 for genome engineering. Cell. 2014 Jun 5;157(6):1262-78. doi:10.1016/j.cell.2014.05.010. Review. PubMed: 24906146. Free full-text available from PubMed Central: PMC4343198.

Komor AC, Badran AH, Liu DR. CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes. Cell. 2017 Apr 20;169(3):559. doi:10.1016/j.cell.2017.04.005. PubMed: 28431253.

Lander ES. The Heroes of CRISPR. Cell. 2016 Jan 14;164(1-2):18-28. doi:10.1016/j.cell.2015.12.041. Review. PubMed: 26771483.

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Bridging The Public Knowledge Gap Around Cell And Gene …

Posted: February 16, 2019 at 10:43 am

By Morrie Ruffin, ARM Foundation for Cell and Gene Medicine

Cell and gene therapies hold the potential to transform medicine; however, if patients are to gain access to these therapies, we must increase public awareness and understanding of how these therapies can benefit them. According to a review of recent research studies that measured U.S. public opinion related to gene medicine,1 the public is not yet aware of gene medicine; is unfamiliar with the terms being used to describe the topic; and, when faced with multiple therapeutic options, is worried about making informed decisions.

The knowledge gap that exists around cell and gene medicine could delay achievement of our shared goal of accelerating the development of potentially curative therapies for a host of life-threatening diseases. One of the top priorities of the ARM Foundation for Cell and Gene Medicine is to increase public awareness and understanding of cell and gene medicine through education projects that highlight the clinical and societal benefits of gene medicine and the life-saving possibilities that these medical advances could bring.

Fearing Unknowns

In April 2016, the New England Journal of Medicine published an analysis of 17 public opinion polls. More than half of those surveyed said they had heard either a little (48 percent) or a lot (9 percent) about gene editing before participating in the survey; 42 percent said they had not heard about gene editing.2 A 2017 Science magazine article concluded there is a broad mandate for public engagement.3 Preliminary research conducted on behalf of the ARM Foundation for Cell and Gene Medicine indicates individuals do not yet see anything fundamentally wrong with gene medicine but are hesitant about its unknowns. People also tell us they are more likely to trust information about gene medicine from a nonprofit organization than the government or pharmaceutical or biotechnology companies.

These findings also mirror a 2014 study, published in Human Gene Therapy, which found that more than 50 percent of respondents said their number one concern was not receiving all the information. According to its authors, delivering effective, evidence-based communications on gene therapy, engaging the research and medical communities as well as the public in a conversation about the ethics of gene therapy, is a priority.4

Call For Clarity

According to our interviewers, research interviewees admit they are confused when people talk about gene therapy. Most struggle to understand the differences in terms, such as somatic and germline gene editing, and concede that a better understanding may affect their opinions of gene medicine. They indicate they would appreciate more information on how gene therapy may be used. One major opportunity for education is information and data suggesting that gene medicine may help treat nonheritable diseases and conditions.

The risk of public misperception is, of course, significant. Gene medicine is advancing at an unprecedented pace with the potential to benefit millions of patients and even the potential to ease the burden of incurable diseases. Public engagement and education, research transparency, and independent level setting based on the facts can help smooth the path to societal acceptance of gene medicine as a viable, nonthreatening treatment option. As we have learned from the research published in Science and other journals as well as our interview-based research, participants who can answer factual questions about gene editing and CRISPR are more in favor of gene therapy.

Moving Gene Medicine Into The Mainstream

The foundation recently launched a strategic communications program designed to generate awareness and support of gene medicine.

The program will inform the public of:

We believe the research is urging us to develop jargon-free content thats easy to access and includes compelling, clear illustrations that summarize the science.

The foundation will support the industry with:

How Gene Therapy Leaders Can Become Involved

Industry and other relevant organizations are invited to become a part of this important education program.

The foundation seeks volunteers to:

The foundation serves as the educational and information catalyst on issues fundamental to making gene and cell therapies, tissue-engineered products, and other regenerative medicine treatments available to patients. By examining, quantifying, clarifying, and informing stakeholders of the clinical and societal benefits of these therapies, as well as convening discussions to raise awareness about the sectors progress and challenges, we can accelerate patient access to safe, efficacious, and potentially curative therapies.

Visit thearmfoundation.org or contact [email protected] for more information.

References:

About The Author:

Morrie Ruffin is co-founder of the Alliance for Regenerative Medicine (ARM) and co-founder, board member and executive director of its 501c3 affiliate, the ARM Foundation for Cell and Gene Medicine. Ruffin is also co-founder and managing partner of Adjuvant Partners, a business development and strategic advisory firm. He has held executive and business development positions at various organizations, including what is now the Biotechnology Innovation Organization (BIO). Earlier, Ruffin worked as a senior legislative assistant for a U.S. senator. He received his MA in international studies and economics from the Johns Hopkins School for Advanced International Studies and his BA from the University of Virginia.

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Immortalitymedicine Website – Immortality Medicine

Posted: February 15, 2019 at 12:43 am

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Posted: February 15, 2019 at 12:43 am

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Immortalitymedicine.tv" Keyword Found Websites Listing …

Posted: February 15, 2019 at 12:43 am

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Posted: February 15, 2019 at 12:43 am

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