Mueller Lab for Gene Therapy

01/13/2023

In a promising proof-of-concept therapeutic approach for amyotrophic lateral sclerosis ( ) and frontotemporal dementia ( ), researchers used an …

04/21/2021

Our efforts have now been advanced by Apic Bio to offer a new hope to patients with SOD1 ALS

Apic Bio, Inc., an innovative gene therapy company developing novel treatment options for patients with rare genetic diseases, today announced that th

07/23/2019

https://www.pharmanewsdaily.com/apic-bio-bags-fda-orphan-designation-for-als-sod1-gene-therapy-apb-102/

US gene therapy company Apic Bio said that APB-102, its ALS SOD1 gene therapy, has been granted orphan drug

03/19/2019

https://theangelfund.org/squirrel-run-xxii/

ABOUT US Our mission is to support ALS (Lou Gehrig’s Disease) research and scientific investigations at the Cecil B. Day Laboratory by raising funds through events, campaigns, foundation grants and numerous other community outreach activities, to aid in finding a cause, treatment and cure for ALS....

03/09/2019

We need you all to vote ! Let’s win this!!

We are excited to share that the two UMass Medical School teams competing in have made it to Round 2. Please take a few minutes to help our researchers remain in the running! http://ow.ly/2Zhz30nYolJ STAT RNA Therapeutics Institute Mueller Lab for Gene Therapy The Angel Fund for ALS Research

03/07/2019

https://www.thesciencebreaker.org/breaks/health-physiology/gene-therapy-and-als-one-step-closer-to-the-clinic

Amyotrophic lateral sclerosis is a devastating, invariably fatal neurodegenerative disorder. Now researchers demonstrate that a novel gene therapy treatment that silences the toxic gene is safe in monkeys. This breakthrough treatment could soon be in the clinic. - submission by Florie Borel, Christi...

01/07/2019

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

Apic Bio, Inc., an innovative gene therapy company developing novel treatment options for patients with rare genetic diseases, today announced the com

05/18/2018

Multiple poster presentations from the lab today at
Poster 655 on gene therapy prevention of alpha-1 antitrypsin emphysema
Poster 898 on ALS

03/15/2018

The Mueller Lab for Gene Therapy is honored to be featured on the cover of PNAS​ issue #11!
"New mouse model of COPD" illustrated by a beautiful (if we may say so ourselves) image of lung alveoli. Thanks again to the University of Massachusetts Medical School​ 's Core Electron Microscopy Facility!
http://www.pnas.org/content/115/11.cover-expansion

02/16/2018

Our latest work is now available online - we describe the first animal model of antitrypsin deficiency.
Alpha-1 Foundation AlphaFriend Maria University of Massachusetts Medical School PNAS
http://www.pnas.org/content/pnas/early/2018/02/15/1713689115.full.pdf

Chronic obstructive pulmonary disease affects 10% of the worldwide population, and the leading genetic cause is a genetic disease, α-1 antitrypsin (AAT) deficiency. Humans have only one gene that codes for the AAT protein, but mice have up to six, which made it impossible for decades to create a mo...

11/01/2017

Our latest work is making the November cover of Molecular Therapy! Proof-of-concept treatment for antitrypsin deficiency. Thanks to our collaborators!

"On the Cover: Diastase-resistant periodic acid-Schiff staining of an untreated PiZ mouse liver. The magenta-colored globules are intracellular inclusions of polymers of the Z-form of α-1 antitrypsin, which is associated with liver pathology. As described in this issue by Borel et al. (pp. 2477–2489), treatment with the AAV-GeneRide-DualFunctionAAT vector reduces the amount of globules-positive hepatocytes by more than 100-fold."
Elsevier Alpha-1 Foundation AlphaFriend Maria University of Massachusetts Medical School

10/23/2017

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

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