Gene Therapy for Degenerative Brain Disorder Wins FDA Nod
The FDA granted accelerated approval to elivaldogene autotemcel (eli-cel; Skysona), a gene therapy to treat early, active cerebral adrenoleukodystrophy (CALD) in boys ages 4 to 17 years, drugmaker bluebird bio announced Friday. Childhood CALD is a rare, neurologically debilitating form of adrenoleukodystrophy that generally occurs in young boys. It is caused by a mutation in […]
CDC: Two Distinct Monkeypox Strains Detected in U.S.
Gene sequencing efforts suggest that two different monkeypox variants are spreading in the U.S., raising the possibility that the virus has been circulating longer than experts initially thought, CDC officials said on Friday. Currently, there have been 20 monkeypox cases identified in 11 U.S. states, all in adults, Jennifer McQuiston, DVM, deputy director of CDC’s […]
T-Cell Receptor Gene Therapy Shows Promise in Pancreatic Cancer
A novel T-cell receptor (TCR) gene therapy may be effective in treating patients with pancreatic cancer and other cancers that express the KRAS G12D mutation, researchers have found. Their case study — published in the New England Journal of Medicine (NEJM) — showed that a heavily pretreated patient with pancreatic ductal adenocarcinoma achieved a deep […]
Gene linked to severe learning disabilities governs cell stress response: Gene associated with Nascimento Syndrome triggers cell’s defenses against environmental attacks
A gene that has been associated with severe learning disabilities in humans has been found to also play a vital role in cells’ response to environmental stress, according to a Duke University study appearing May 24 in the journal Cell Reports. Cells are stressed by factors that may damage them, such as extreme temperatures, toxic […]
A ‘factory reset’ for the brain may cure anxiety, drinking behavior, study suggests: Gene editing reverses brain genetic reprogramming caused by adolescent binge drinking
Gene editing may be a potential treatment for anxiety and alcohol use disorder in adults who were exposed to binge drinking in their adolescence, according to the results of an animal study published in the journal Science Advances. The study is issued by researchers from the University of Illinois Chicago who have been studying the […]
New path to treat advanced triple-negative breast cancer
A new study by researchers at Yale Cancer Center shows inhibition of the CECR2 gene prevents triple-negative breast cancer from advancing or metastasizing. The discovery is an early step in finding new therapeutics for triple-negative breast cancer (TNBC), one of the most difficult disease sub-types to treat. The findings are published online today in the […]
New findings on the link between CRISPR gene-editing and mutated cancer cells
A protein that protects cells from DNA damage, p53, is activated during gene editing using the CRISPR technique. Consequently, cells with mutated p53 have a survival advantage, which can cause cancer. Researchers at Karolinska Institutet in Sweden have found new links between CRISPR, p53 and other cancer genes that could prevent the accumulation of mutated […]
Regular Blood Work After Gene Therapy Warranted by Rare Condition
Recipients of certain adeno-associated virus (AAV) vector-based gene therapies should be monitored closely for signs of thrombotic microangiopathy (TMA), FDA advisors agreed. “Frequent laboratory assessments are necessary,” said Barry Byrne, MD, PhD, of the University of Florida in Gainesville, based on his observations that patients on onasemnogene abeparvovec (Zolgensma) tend to first show IgM presence […]