Source:https://www.medicalnewstoday.com/articles/327296.php Using a new type of genetic engineering tool called epigenome editing in mice, scientists have restored irregularities in the developing brain that arise from a gene mutation. New research in mice suggests that gene editing could prevent brain developmental disorders.Epigenome editing is a way of altering the expression, or reading, of genes without altering their underlying DNA code. A team from Johns Hopkins University in Baltimore, MD, led the Nature Communications study that focuses on the protein C11orf46. One of the study’s corresponding authors is Dr. Atsushi Kamiya, who is an associate professor of psychiatry and behavioral sciences at Johns Hopkins University School of Medicine. In humans, mutations in the section of DNA that contains the C11orf46 gene can lead to WAGR syndrome, a genetic condition that can cause intellectual disability and impair many systems of the body. The researchers found that C11orf46 directs the development of the corpus ...
Prevail Therapeutics, Inc. (Prevail), a biotechnology company developing gene therapies for patients with Parkinson’s disease and other neurodegenerative diseases, today announced a $75 million Series A financing.
Gene therapy has freed 10 men from nearly all symptoms of hemophilia for a year so far, in a study that fuels hopes that a one-time treatment can give long-lasting help and perhaps even cure the blood disease.
As is well known, CRISPR/Cas9 is an effective tool for gene editing, and this method has been widely used for editing genes or genomic regions by targeting specific single-guide RNA (sgRNAs). We initially planned to use the CRISPR/Cas9 system to study the biological functions of Y chromosome genes.
Obesity is often attributed to a simple equation: People are eating too much and exercising too little. But evidence is growing that at least some of the weight gain that plagues modern humans is predetermined.
Authorities in the city of Nanjing, the capital of China’s Jiangsu province, announced at the end of October that they would sequence the genes of 1 million individuals to build a genetic database of Chinese residents. This project is part of the National Health and Medicine Big Data Nanjing Center, a new data storage facility under construction in the region.
A vaccine combining centralized ancestral genes from four major influenza strains appears to provide broad protection against the dangerous ailment, according to new research by a team from the Nebraska Center for Virology.
The NHS will fund gene therapy for the first time, with a £500,000 treatment for “bubble baby syndrome”.
Gene therapy is designed to deliver a one-off cure for the patient and drugmakers are typically asking a hefty price that is comparable to the combined costs of alternative life-long treatment.
Spark Therapeutics Inc's experimental gene therapy for a rare form of blindness improves vision and should be approved, advisers to the Food and Drug Administration concluded on Thursday, paving the way for the first U.S. gene therapy for an inherited disease.
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