Mueller Lab for Gene Therapy

American Society of Gene & Cell Therapy on LinkedIn: CRISPR/Cas9 Gene Editing Used to ‘Cut Out’ ALS Mutation: Early... In a promising proof-of-concept therapeutic approach for amyotrophic lateral sclerosis ( ) and frontotemporal dementia ( ), researchers used an …

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

Apic Bio Announces FDA Clearance of IND Application for Lead Gene Therapy Candidate APB-102 for the Treatment of SOD1 ALS Apic Bio, Inc., an innovative gene therapy company developing novel treatment options for patients with rare genetic diseases, today announced that th

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

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

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

The Angel Fund for ALS Research | An Independent Non-Profit Charity 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....

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

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

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...

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

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

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

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 PNAShttp://www.pnas.org/content/pnas/early/2018/02/15/1713689115.full.pdf

Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema 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...

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

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.