Nature

The Wild West of spike-in normalization

The proper use of spike-in normalization in ChIP-seq improves sensitivity for detecting genome-wide changes between conditions, but improper use is common, calling some biological conclusions into ...
Nature

Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice

Durable factor VIII expression that normalizes hemostasis is an unrealized goal of hemophilia A adeno-associated virus-mediated gene therapy. Trials with initially normal factor VIII activity observed ...
The New England Journal of Medicine

Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B

FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a synthetic capsid and a gain-of-function protein to normalize factor IX levels in patients ...
Healio

Novel gene therapy reduces bleeding events, need for factor IX infusions in hemophilia B

Please provide your email address to receive an email when new articles are posted on . A single infusion of verbrinacogene setparvovec resulted in normal factor IX levels among all 10 men who ...