Gene Therapy

FDA approves $2.1 million Novartis' 'Zolgensma', makes it the costliest drug ever

Zolgensma, a one-time drug for spinal muscular atrophy, affects 1 in 10,000 children and mostly leads to death.

World's first gene therapy treatment to halt common, untreateable form of blindness

Scientists are using the natural infective ability of a virus to deliver the treatment to patients' cells.

Gene editing in humans shows promising early results in a historic first attempt

Patients who underwent the therapy may not need other treatment for the rest of their lives

CRISPR gene-editing successfully stops muscle-eating disease in young dogs

Cutting out a tiny part of the faulty gene recovered enough of the proteins normal function

Gene therapy: FDA eases restrictions on the once-exotic means of treatment

“Tools we use to address other areas of science are now well suited to gene therapy,” the study reads.

Fighting arthritis may be easier thanks to SMART cells built on CRISPR gene-editing tool

Using the gene-editing tool CRISPR, researchers have found a way to rewire mouse stem cells to fight inflammation caused by arthritis and other chronic conditions.

Angelina’s breast surgery possible in India, but there's a catch

So can we test in India and gain from the same treatment privilege that Jolie had? Yes, but there is a catch.