Mueller Lab

Excited to share our discovery of a new programmable RNA-guided DNA-targeting system hiding inside bacteriophages that predates CRISPR. We call it VIPR (Viral Interference Programmable Repeat), and it uses an entirely new logic to find its targets. Thread + link below.

It just seems implausible this is what we are made of, essentially, nanotechnology about a billion years beyond anything we can design or make ourselves.

Today in @ScienceMagazine we report the development of a laboratory-evolved CRISPR-associated transposase (evoCAST) that supports therapeutically relevant levels of RNA-programmable gene insertion in human cells, a collaboration with @SternbergLab. 1/13 drive.google.com/file/d/1I-UbCR…

Ambros and Ruvkun’s #NobelPrize -winning #miRNA discovery has provided another tool in the #genetherapy arsenal! linkedin.com/posts/drchrism…

A door into a new era of physics has just opened up! youtu.be/uOJCS1W1uzg

Proud to share the latest work on Base Editing for Alpha-1 ⁦@AlphaFriend⁩ ⁦@alpha1bfc⁩ linkedin.com/posts/drchrism…

Dual-function AAV vector to prevent alpha-1 antitrypsin deficiency @muellerlab at UMass Medical School has developed a dual function approach using AAV #vectors targeting both #lung and #liver #diseases. Find out more: bit.ly/3msB6bL

An Ultimate Mouse Model of #Gene #Therapy for Alpha1 Anti-trypsin Deficiency @muellerlab led by Dr Dr. Marina Zieger, Ph.D. bit.ly/385d52U

To this day I get replies and DMs saying, “give up, you can’t end ALS.” Let me make this simple: I have ALS. ALS is currently terminal. It does not have to be terminal. We’re on the precipice of change. I will be part of making that change real and ending ALS. Any questions?

A rewarding culmination of my 20 year academic career in #AAV #genetherapy ! I look forward to maturing what I incubated in the academic sector in the private sector to hopefully bring gene therapies to market and help patients with #rarediseases bit.ly/2Ce2wQ2

Muscle-Directed Delivery of an #AAV Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans: Molecular Therapy #GeneTherapy cell.com/molecular-ther…

Dual-function #AAV vector to prevent ⁦@AlphaFriend⁩ -1 antitrypsin deficiency ⁦@UMassMedical⁩ ⁦@UMassMedRTI⁩ #COPD youtube.com/watch?v=at_dLF…

The @muellerlab at UMass Medical School has developed a #gene transfer approach that could be the future of ALS #treatment by silencing the #mutated SOD1 gene using microRNAs. youtube.com/watch?v=gRoO_F…

@RitaBat82 Your not just part of the team. Your part of the foundation the team got built on!

Proud of being part of this team!

JONATHAN YOU CAN DO IT, WE CAN DO IT! Like and RT! Jonathan Maidment, a 24-year-old Alpha is about to finish his hike of the entire 2,650 miles of the Pacific Crest Trail. Help him get 2,650 LIKES on this pic to get a $20K donation towards his goal! #Hiking4ACure #Alpha1Awareness

Thanks to our Apic Bio team of riders and volunteers who participated in the annual Escape to the Cape Bike Trek 2019. Together we raised awareness for Alpha-1 and showed our dedication to #raredisease! #ridingforareason #curealpha1 #Alpha1 #GeneTherapy

Beautiful Day at the Cape supporting our riding for our @AlphaFriend #COPD #alpha1