Mueller Lab for Gene Therapy

Apic is helping move our lab’s great work into the clinic!

Apic Bio Announces $40M Series A Financing to Advance Novel Gene Therapies for Rare Genetic Disorders Apic Bio, Inc., an innovative gene therapy company developing novel treatment options for patients with rare genetic diseases, today announced the com

The Mueller Lab for Gene Therapy has a new publication coming out in November in the journal Molecular Therapy. This work is the first proof of concept of a approach for the treatment of antitrypsin deficiency. It was featured last week on UMassMedNow.
“What we have here is a proof of concept that this approach would potentially help patients,” said Mueller. “And for very young patients with actively growing livers that could potentially be treated early in life, this could be very meaningful.”
Elsevier University of Massachusetts Medical School AlphaFriend Maria Alpha-1 Foundation

New gene editing approach for alpha-1 antitrypsin deficiency shows promise in UMMS study UMass Medical School study by Mueller lab finds promise in "nuclease-free" gene editing for alpha-1 antitrypsin deficiency.