Jennifer A. Doudna and Emmanuelle Charpentier were awarded the 2020 Nobel Prize for Chemistry for their discovery of a technology, described by the Nobel Committee as “Genetic scissors: a tool for rewriting the code of life”. CRISPR (a wisely chosen acronym for Clustered Regularly Interspaced Short Palindromic Repeats) in combination with the enzyme Cas9 (CRISPR-associated protein 9), is a new breakthrough technology that can target a section of DNA, literally cut out a sequence of interest and insert a new sequence in its stead that is then incorporated during the process of DNA repair. There is no doubt that CRISPR/Cas9 represents a watershed moment in science, and applications for therapeutics and even cures of disorders once thought incurable are on the horizon. The Nobel Committee appropriately describes CRISPR as a “technology has had a revolutionary impact on the life sciences, is contributing to new cancer therapies and may make the dream of curing inherited diseases come true.”
In a publication by Ma et al. (Nature, 2017), researchers were able to take human preimplantation embryos and cut out and replace a disease-causing pathogenic variant, using the CRISPR/Cas9 gene-editing tool.
In anticipation of a fast-paced research trajectory, The American Society of Human Genetics (ASHG) brought together an international workgroup that included representatives from the following organizations
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The following positions are stated in this document:
(1) At this time, given the nature and number of unanswered scientific, ethical, and policy questions, it is inappropriate to perform germline gene editing that culminates in human pregnancy. (2) Currently, there is no reason to prohibit in vitro germline genome editing on human embryos and gametes, with appropriate oversight and consent from donors, to facilitate research on the possible future clinical applications of gene editing. (3) There should be no prohibition on making public funds available to support this research. (4) Future clinical application of human germline genome editing should not proceed unless, at a minimum, there is (a) a compelling medical rationale, (b) an evidence base that supports its clinical use, (c) an ethical justification, and (d) a transparent public process to solicit and incorporate stakeholder input.
The ObG Project highly recommends you join the almost 2-million people who have viewed Dr. Jennifer Doudna’s TED talk. As a co-inventor of CRISPR/Cas9, she explains this groundbreaking technology in less than 20 minutes and makes the case for why this research must be done within an ethical framework.
How CRISPR lets us edit our DNA | Jennifer Doudna
Correction of a pathogenic gene mutation in human embryos
ASHG Position Statement: Human Germline Genome Editing
Review: Development and Applications of CRISPR-Cas9 for Genome Engineering
Review: CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes
Development of CRISPR as an Antiviral Strategy to Combat SARS-CoV-2 and Influenza
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