Mueller Lab

Our Lab is dedicated to studying rare inherited disorders and translating gene centered therapies to treat them. #Alpha1, #ALS, #CF, #HD, #HPS

[Kenneth Loi @kenjmloi]

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.

[Andrew Côté @Andercot]

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.

[David R. Liu @davidrliu]

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
https://t.co/ZLHtZm5Gmw

[Mueller Lab @muellerlab]

Ambros and Ruvkun’s #NobelPrize -winning #miRNA discovery has provided another tool in the #genetherapy arsenal! https://t.co/At6AJj1JTS

[Mueller Lab @muellerlab]

https://t.co/7OzgRgOx1E

[Mueller Lab @muellerlab]

A door into a new era of physics has just opened up! https://t.co/ydc1iJp5RW

[Mueller Lab @muellerlab]

Proud to share the latest work on Base Editing for Alpha-1 ⁦@AlphaFriend⁩ ⁦@alpha1bfc⁩ https://t.co/xLZhIdgco3

[Mueller Lab @muellerlab]

https://t.co/KNW8cAXtAp

[Science Animated @Sci_Ani]

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:

https://t.co/BdzuKCtEL7

[Science Animated @Sci_Ani]

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

https://t.co/SdwBf2LLeJ

[Brian Wallach @bsw5020]

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?

[Mueller Lab @muellerlab]

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 https://t.co/jVwNUs5Wqk

[Mueller Lab @muellerlab]

Muscle-Directed Delivery of an #AAV Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans: Molecular Therapy #GeneTherapy https://t.co/ylwAQsdrmQ

[Mueller Lab @muellerlab]

Dual-function #AAV vector to prevent ⁦@AlphaFriend⁩ -1 antitrypsin deficiency ⁦@UMassMedical⁩ ⁦@UMassMedRTI⁩ #COPD https://t.co/iru93StRix

[Science Animated @Sci_Ani]

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.

https://t.co/ABitKoOfHn

[Mueller Lab @muellerlab]

https://t.co/v1J2gJljja

[Mueller Lab @muellerlab]

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

[Alpha-1 Foundation @AlphaFriend]

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

[Apic Bio @ApicBio]

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

[Mueller Lab @muellerlab]

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