New Tool Identifies Safe Places To Introduce Genes Into Human DNA
Scientists at St. Jude Children’s Research Hospital have developed a tool that can locate safe places to introduce genes into human DNA. The tool is an…
Gene Therapy News
Gene Therapy Can Restore Night Vision After Decades of Congenital Blindness
The patients had Leber Congenital Amaurosis, a type of genetic congenital blindness. According to researchers at the Scheie Eye Institute at the Perelman School of…
More Potent, Less Toxic: Scientists Develop a Better Type of Chemotherapy
Researchers discover a new class of drugs that provide leukemia patients with a safer, more targeted form of therapy. Chemotherapy isn’t fun. It’s no secret…
Gene Therapy Successfully Restores Cone Function in Colorblind Children
Following treatment, two of the four children’s measurements of cone function closely resembled those of normal-sighted research participants. According to a recent study led by…
Potentially Hazardous: CRISPR Therapeutics Could Promote Cancer
The study showed that CRISPR therapeutics can damage the genome. The researchers caution: “The CRISPR genome editing method is very effective, but not always safe….
Genetic Testing Could Help People Suffering From Depression
Positive results from a study on precise medication dosing. According to a recent research conducted by the U.S. Department of Veterans Affairs, pharmacogenomic testing may assist…
New Hope for Hearing Loss Treatment: Researchers Identify 48 Genes Linked to Hearing Loss
Researchers identified 48 genes that are associated with hearing loss, including 10 new variants. Hearing loss is a common problem caused by noise, age, disease,…
A Potential Cure for Autism: New Research Finds That Gene Therapy Could Treat Pitt-Hopkins Syndrome
New research from the UNC Neuroscience Center lab of Ben Philpot, Ph.D., finds restoring lost gene activity prevents many disease signs in an animal model…
A New Way That Cancer Cells Can Repair DNA Damage Has Been Discovered
Identifying DNA Repair Genes Holds Promise for Improving Cancer Treatment Cancer is one of the leading causes of death in the United States and worldwide….
Gene Therapy Successfully Treats Spinal Cord Injuries Without Side Effects
In mouse studies, pain-blocking neurotransmitters produced long-lasting benefits without detectable side effects. An international team of researchers led by scientists at the University of California…
Gene Therapy Reverses Effects of Autism-Linked Mutation in Human Brain Organoids
University of California San Diego (UCSD) study uses lab-grown human brain tissue to identify neural abnormalities in Pitt-Hopkins Syndrome and test gene therapy tools. In…
“Time Jump” by 30 Years: Old Skins Cells Reprogrammed To Regain Youthful Function
Findings could lead to targeted approach for treating aging. Research from the Babraham Institute has developed a method to ‘time jump’ human skin cells by…
Tweaked Genes Borrowed From Bacteria Electrically Excite Heart Cells in Live Mice
First approach to promote electrical excitation of heart cells in live mammals could lead to new gene therapy treatments for a wide range of heart…
Landing Therapeutic Genes Safely in the Human Genome – Improving Gene and Cell Therapies
Researchers at Harvard’s Wyss Institute, Harvard Medical School, and the ETH Zurich predict and validate genomic safe harbors for therapeutic genes, enabling safer, more efficient,…
Over 4 Million Deaths per Year Caused by Obesity: Safe, Non-Invasive Treatments Could Help End Epidemic
Novel obesity treatments such as modulation of the gut microbiome and gene therapy are underutilized and could help fight the obesity epidemic, according to a…
Researchers Engineer a Mini CRISPR Genetic Editing System That Could Be Easier To Deliver Into Cells
A compact and efficient CRISPR-Cas system, named CasMINI, could be broadly useful for cell-engineering and gene-therapy applications because it is easier to deliver into cells….
“Mini” CRISPR Genetic Editing System Engineered – Easier To Deliver Into Human Cells for Gene Therapy
Bioengineers have repurposed a “non-working” CRISPR system to make a smaller version of the genome engineering tool. Its diminutive size should make it easier to…
New Gene Therapy Developed That Could Be Effective Against Many Types of Cancer
Mount Sinai researchers have developed a therapeutic agent that shows high effectiveness in vitro at disrupting a biological pathway that helps cancer survive, according to…
