Crispr

CHICAGO — A CRISPR-Cas9-based therapy targeting the gene encoding transthyretin reduced transthyretin levels in patients with cardiac amyloidosis, researchers reported at the American Heart Association Scientific Sessions.

It is almost impossible to pick up a medical journal today without seeing an article or commentary on gene editing.

SEATTLE — In this video, Aleksandra V. Rachitskaya, MD, FASRS, discusses some of the key highlights in neovascular age-related macular degeneration from the ASRS annual meeting.

Two teenagers with T-cell acute lymphoblastic leukemia achieved complete molecular remission after receiving a donor-based investigational chimeric antigen receptor T-cell therapy, phase 1 study results showed.

LOUISVILLE, Ky. — All patients treated with one infusion of NTLA-2002 gene therapy have remained hereditary angioedema attack-free, according to data presented at American College of Allergy, Asthma & Immunology Annual Scientific Meeting.

CHICAGO — A novel CRISPR-based in vivo gene editing therapy reduced transthyretin levels in patients with hereditary transthyretin amyloidosis with cardiomyopathy, researchers reported at the American Heart Association Scientific Sessions.

The advent of novel therapies has propelled, and continues to propel, tremendous progress and innovation in diffuse large B-cell lymphoma.

With the aid of $17 million in grant funding, UCSF Benioff Children’s Hospital Oakland will initiate a 4-year trial evaluating CRISPR/Cas9 gene editing as a potential cure for sickle cell disease.

The FDA granted orphan drug designation to NTLA-5001 for treatment of adults with relapsed or refractory acute myeloid leukemia.