Researchers say findings establish HOX genes as potential therapeutic targets for enhancing bone regeneration.

A new study finds that the VRC01 antibody initially protects against HIV infection, but resistant strains can cause breakthrough infections.

Many of the blood proteins that could flag neurodegeneration are so scarce that standard assays barely register them. A new UK Biobank study will test whether an ultrasensitive platform can pick them ...

The findings show precise correction of the SCD mutation in the β-globin gene in three SCD patients as well as restored expression of adult hemoglobin (HbA).

In this GEN eBook readers will discover how epigenomic insights are improving every stage of the drug discovery pipeline, ...

Findings may offer insights into disorders associated with dysregulated forgetting, including dementia and PTSD.

The FDA has set a May 1 target action date for uniQure’s Huntington’s disease gene therapy candidate ifezuntirgene inilparvovec, after accepting the company’s Biologics License Application (BLA) under ...

The FDA has delayed a decision on Agios Pharmaceuticals’ supplemental New Drug Application seeking additional approval for mitapivat in sickle cell disease until next year.

Study findings reframe pulmonary hypertension as a multi-organ inflammatory disorder and point to potential diagnostic, prognostic targets.

Genomics launches Sentira, an Anthropic Claude-powered AI platform designed to accelerate single-cell data analysis.

Single-cell atlas gives researchers a baseline for exploring how injury, aging, drugs, or other changes affect cells across ...

Patient safety, expanding access to gene therapy, and progress towards an HIV vaccine were prominent themes among the early ...