clinical trials, genetics & genomics

Four dozen children with severe combined immunodeficiency (SCID) who received a corrective gene carried by a virus have working immune systems two to three years later, according to three independent clinical trials.

It’s not clear why the patients with Leber hereditary optic neuropathy, a mitochondrial disorder that causes blindness, also experienced the modest benefits in their untreated eye.

The University of Alabama at Birmingham neuroscientist aims to determine which cells are most important in prompting the disease’s initiation and progression.

The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.

Despite a solid understanding of the biological basis of fragile X syndrome, researchers have struggled to develop effective treatments.

Take a break from the bench to puzzle and peruse.

Two studies show signs that the introduced DNA is functioning, but it’s too early to know if patients actually benefit.

Two patients who received Sangamo’s zinc finger–based treatment for Hunter syndrome have lower biomarkers of the condition, but no signs of new enzyme production.

The Germany-based study will test an ex vivo genome-editing therapy for the inherited blood disorder β-thalassemia.

The agency proposes ceding its scrutiny of these studies to the FDA.

The medicine increases the activity of a deficient enzyme in certain patients with the condition.

The results could lead to a treatment to lower cholesterol in patients with hypercholesterolemia.

Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.

The 44-year-old patient has Hunter syndrome, which doctors hope to treat using zinc finger nucleases.

The publicly available database found nearly a third of samples included mutations targeted by either approved drugs or therapies in clinical trials. 

A screen of human embryonic stem cell lines finds several that accumulated changes in the gene TP53, including aberrations commonly seen in cancer.

At the annual American Association for Cancer Research meeting, researchers discuss the importance of understanding the epigenetic contributors to cancer progression and treatment response.

Sorting out which data sets are clinical-grade is key to helping patients.

The results of a Phase 2 trial suggest that delivering normal copies of the gene that causes cystic fibrosis may slow lung decline.