In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs ..read more

Researchers at Washington University School of Medicine in St. Louis have received a $6.2 million grant from the National Institutes of Health (NIH) to develop a gene therapy that would modify the immune system's B cells to spur them to produce broadly neutralizing antibodies against HIV ..read more

The most common type of lung fibrosis -; scarring of the lungs -- is idiopathic, meaning of unknown cause ..read more

Fluent BioSciences, a cutting-edge life sciences company leading the charge in revolutionizing single-cell analysis through simple, cost-effective, and highly scalable single-cell RNA sequencing solutions is thrilled to announce the award of an NIH Small Business Innovation Research (SBIR) Phase II grant, funded by the National Institute of General Medical Sciences (NIGMS, 2 R44 GM137648 ..read more

Argininosuccinate lyase deficiency (ASLD), also known as argininosuccinic aciduria, is a disease that has been enriched in the Finnish genetic heritage ..read more

Weill Cornell Medicine scientists have developed an innovative human neuron model that robustly simulates the spread of tau protein aggregates in the brain-;a process that drives cognitive decline in Alzheimer's disease and frontotemporal dementia ..read more

Since its breakthrough development more than a decade ago, CRISPR has revolutionized DNA editing across a broad range of fields ..read more

RS Genomics Limited, which was formed to provide broad access to the foundational CRISPR/Cas9 intellectual property co-owned by Dr. Emmanuelle Charpentier, today announced its patent CN201380038920.6 was upheld by the CNIPA in response to an invalidation challenge ..read more

Messenger RNA (mRNA) made its big leap into the public limelight during the pandemic, thanks to its cornerstone role in several COVID-19 vaccines ..read more

Although there are many differences between type 1 diabetes and type 2 diabetes, there are also similarities, such as inflammation of the insulin-producing cells ..read more