National Institute of Health announces the results of research showing that genetic editing techniques can safely be used to treat hereditary diseases

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In "genome editing" which has a sense of feeling that stepped into the realm of God that "you can freely design genes to change the characteristics of organisms", you can manipulate genes to create ideal crops and organisms, It is expected to treat it. Meanwhile, arguments have been raised about improving human genes, but Dr. Francis Collins, the director of the National Institutes of Health, has published a self-directed blog with a safe gene using DNA editing technology We are reporting the research results showing the prospect of treatment.

Find and Replace: DNA Editing Tool Shows Gene Therapy Promise | NIH Director's Blog
https://directorsblog.nih.gov/2017/01/24/find-and-replace-dna-editing-tool-shows-gene-therapy-promise/

In the field of gene therapy research, methods for safely inserting copies of normal genes into people with genetic defects are being sought. In recent years, technologies for editing such genes have been progressing, and it has come to be known as "genome editing" that enables genetic manipulation at an order of magnitude faster than traditional genetic engineering techniques. You can understand the differences between the genes, DNA and genome in the first place and what kind of technology is "genome editing" by reading the following article.

What technology is "genome editing" in God's domain that can freely design genes and change the characteristics of living things? - GIGAZINE


In the blog of Dr. Francis Collins, Director of the National Institutes of Health, "Chronic granulomatosisResearch on gene therapy for immunodeficiency disease has been reported. Chronic granulomatosis is "Hematopoietic stem cells"The research team of the National Institutes of Health uses the latest genomic editing technology" CRISPR / Cas9 "that cuts a specific gene and incorporates another gene, and it is caused by the mutation of hematopoietic stem cells We have found a cure that can modify the mutation and do not potentially cause another disease.

The team conducted an experiment of transplanting edited human cells to mice, and it was confirmed that it was established in the bone marrow and produced leukocytes that function completely. Bone marrow with murine cells modified by gene editing succeeded in flowing normal blood flow for 5 months. This indicates that we can establish a new therapy someday without any risks or restrictions than "traditional hematopoietic stem cell transplant" which is a treatment for chronic granulomatosis.

In addition, the National Institutes of Health receives approval for clinical trial application to humans using editing technology "CRISPR / Cas 9" in June 2016.

Clinical trial applications using genomic editing technology "CRISPR / Cas9" for human genetic manipulation will be approved - GIGAZINE


Despite the results of experiments using mice this time, safe gene therapy methods for humans are being put to practical use. Dr. Collins of the National Institutes of Health said, "This news is a promising sign that we can treat various hereditary diseases, not limited to chronic granulomatosis." In addition, the discussion on "How much should you add to human genes?" Is explained in the following article.

What is the genetic editing technology "CRISPR" and what is the future of humanity and humanity? - GIGAZINE