The Dickey-Wicker amendment prohibits the use of Congressionally appropriated funds for the creation of human embryos for research purposes or for research in which human embryos are destroyed. NIH does not fund any use of genome editing in human embryos. Ethical and Safety ConcernsĪpplications of genome editing have raised scientific, safety, ethical and policy concerns. In October 2019, NIH and the Bill and Melinda Gates Foundation announced a collaboration to support studies to advance the development of gene-based approaches to cure sickle cell disease and HIV. Genome editing approaches are also being pursued as part of NIH’s Cure Sickle Cell Initiative, and CRISPR is being used as a diagnostic tool to detect viruses such as Zika and dengue.
TALENs are being studied in T cell immunotherapy approaches to create “off-the-shelf” universal donor T cells that don’t have to be developed for each cancer patient. The clinical trial was the first genome editing approach administered directly to research participants. Caused by an enzyme deficiency, Hunter syndrome can cause abnormalities in the skeleton, heart and respiratory system. In 2017, a clinical trial testing ZFNs to correct Hunter syndrome (MPS II) was launched. In 2014, the first clinical application of genome editing involved the use of ZFNs to make human cells resistant to HIV-1 by disrupting a gene required for the virus to infect cells. CRISPR and other gene editing methods, especially ZFNs, are speeding gene therapy approaches to treat many human conditions. This means that changes in somatic cells are not inherited by subsequent generations.
Somatic cells are any cells not involved in human reproduction. NIH supports human gene therapy research, including genome editing approaches in somatic cells, for a wide array of diseases and conditions with grants, contracts and targeted efforts, such as the Somatic Cell Genome Editing Program. This research could be applied to alter mosquito populations to help interfere with the transmission of infectious diseases. Gene drive technology using CRISPR allows for the spread of engineered traits through populations of sexually reproducing organisms at a rate more rapid than what occurs in natural evolution. It also is being explored to modify yeast cells to make biofuels and to improve strains of agricultural crops. It also is being used to change genes in certain tissues or organs, facilitate the study of diseases by focusing on culprit genes, create cell models of disease such as in human pluripotent stem cells and inactivate viruses in pigs so that pig organs could potentially be used as a source of replacement organs for humans. For example, CRISPR is used to make “knockout” models of disease in a wide range of animals, enabling researchers to study the underlying genetic causes. Genome editing is widely used in studies in a variety of organisms. CRISPR/Cas9 works by cutting a DNA sequence at a specific genetic location and deleting or inserting DNA sequences, which can change a single base pair of DNA, large pieces of chromosomes, or regulation of gene expression levels.
CRISPR was discovered through NIH-funded basic research on how bacteria defend themselves from viruses. Because CRISPR/Cas9 is an RNA-based system, it can be more efficiently and easily modified than the protein-based approaches and allows for targeting of multiple sites. An additional method is called clustered regularly interspaced short palindromic repeats, also known as CRISPR/Cas9.ĬRISPR/Cas9 is the most widely used genome editor and is a powerful tool for understanding gene function. Genome editing allows researchers to mimic this natural process of DNA repair.Īdvanced genome editing methods engineered from proteins include zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and meganucleases. Genome editing builds on an earlier discovery that a broken section of DNA in a gene triggers a cell’s repair mechanism to stitch together the break. While techniques to modify DNA have existed for several decades, new methods have made genome editing faster, cheaper and more efficient. Genome editing can be used to correct, introduce or delete almost any DNA sequence in many different types of cells and organisms. Genome editing, also called gene editing, is an area of research seeking to modify genes of living organisms to improve our understanding of gene function and develop ways to use it to treat genetic or acquired diseases. Genome Editing in Research Genome Editing in the Clinic Ethical and Safety Concerns Genome Editing and NIH Funding Images Video Additional Resources
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