SCGE
@scge.bsky.social
The Somatic Cell Genome Editing (SCGE) Consortium is an NIH Common Fund program that aims to develop safe and effective methods to perform gene editing to treat genetic diseases in somatic cells. Reposts/likes do not equal endorsements.
scge.mcw.edu
scge.mcw.edu
Kiran Musunuru and Sonia Vallabh, panelists on "Concluding Panel: The Future of Targeted In Vivo Gene Editing" (3/3)
November 21, 2025 at 3:56 PM
Kiran Musunuru and Sonia Vallabh, panelists on "Concluding Panel: The Future of Targeted In Vivo Gene Editing" (3/3)
Rebecca Ahrens-Nicklas, "Personalized liver-directed corrective gene editing" (2/3)
November 21, 2025 at 3:56 PM
Rebecca Ahrens-Nicklas, "Personalized liver-directed corrective gene editing" (2/3)
Are you at the @asgct.bsky.social Breakthroughs in Targeted In Vivo Gene Editing event? Hear from these SCGE researchers on Friday:
Sonia Vallabh, "AAV-mediated epigenetic editing for prion disease" (1/3)
Sonia Vallabh, "AAV-mediated epigenetic editing for prion disease" (1/3)
November 21, 2025 at 3:56 PM
Are you at the @asgct.bsky.social Breakthroughs in Targeted In Vivo Gene Editing event? Hear from these SCGE researchers on Friday:
Sonia Vallabh, "AAV-mediated epigenetic editing for prion disease" (1/3)
Sonia Vallabh, "AAV-mediated epigenetic editing for prion disease" (1/3)
Krishanu Saha, "Genome-wide CRISPR screening identifies cellular factors controlling nonviral genome editing efficiency" (Poster)
@sahakris.bsky.social (4/4)
@sahakris.bsky.social (4/4)
November 20, 2025 at 1:51 PM
Krishanu Saha, "Genome-wide CRISPR screening identifies cellular factors controlling nonviral genome editing efficiency" (Poster)
@sahakris.bsky.social (4/4)
@sahakris.bsky.social (4/4)
Yong-hui Jiang, "Brain-wide genome editing via STEP-RNPs for treatment of Angelman syndrome" (3/4)
November 20, 2025 at 1:51 PM
Yong-hui Jiang, "Brain-wide genome editing via STEP-RNPs for treatment of Angelman syndrome" (3/4)
Kiran Musunuru, "LNP-mediated editing for treatment of cardiometabolic disease"
@kiranmusunuru.bsky.social (2/4)
@kiranmusunuru.bsky.social (2/4)
November 20, 2025 at 1:51 PM
Kiran Musunuru, "LNP-mediated editing for treatment of cardiometabolic disease"
@kiranmusunuru.bsky.social (2/4)
@kiranmusunuru.bsky.social (2/4)
Are you attending American Society of Gene & Cell Therapy's Breakthroughs in Targeted In Vivo Gene Editing event? If so, check out presentations from these SCGE researchers on Thursday:
Bill Peranteau, "In utero editing of muscles with LNPs" (1/4)
@asgct.bsky.social
Bill Peranteau, "In utero editing of muscles with LNPs" (1/4)
@asgct.bsky.social
November 20, 2025 at 1:51 PM
Are you attending American Society of Gene & Cell Therapy's Breakthroughs in Targeted In Vivo Gene Editing event? If so, check out presentations from these SCGE researchers on Thursday:
Bill Peranteau, "In utero editing of muscles with LNPs" (1/4)
@asgct.bsky.social
Bill Peranteau, "In utero editing of muscles with LNPs" (1/4)
@asgct.bsky.social
The SCGE platform now has the first sets of researchers' regulatory documents available to the public. These documents are from regulatory interactions that SCGE researchers have had with the U.S. Food and Drug Administration (FDA).
Access these documents at: scge.mcw.edu/platform/pub...
Access these documents at: scge.mcw.edu/platform/pub...
November 5, 2025 at 2:36 PM
The SCGE platform now has the first sets of researchers' regulatory documents available to the public. These documents are from regulatory interactions that SCGE researchers have had with the U.S. Food and Drug Administration (FDA).
Access these documents at: scge.mcw.edu/platform/pub...
Access these documents at: scge.mcw.edu/platform/pub...
Clinicians want to be informed about clinical trials so they can offer the most up-to-date information to their patients. But with so many trials and busy clinic days, it can be difficult to find the time to read everything.
September 24, 2025 at 5:44 PM
Clinicians want to be informed about clinical trials so they can offer the most up-to-date information to their patients. But with so many trials and busy clinic days, it can be difficult to find the time to read everything.
Closing remarks from Dr. Joni Rutter.
September 17, 2025 at 6:42 PM
Closing remarks from Dr. Joni Rutter.
Speakers, continued (2/2):
- Xiaona Lu, Yale University
- Nicole Vo, University of Washington
- Christy George, University of California, Berkeley
- Xiaona Lu, Yale University
- Nicole Vo, University of Washington
- Christy George, University of California, Berkeley
September 17, 2025 at 3:11 PM
Speakers, continued (2/2):
- Xiaona Lu, Yale University
- Nicole Vo, University of Washington
- Christy George, University of California, Berkeley
- Xiaona Lu, Yale University
- Nicole Vo, University of Washington
- Christy George, University of California, Berkeley
We had some excellent talks by trainees and young investigators this morning! These speakers include (1/2):
- Sarah Grandinette, University of Pennsylvania
- Xiaoshu Pan, University of Florida
- Sarah Grandinette, University of Pennsylvania
- Xiaoshu Pan, University of Florida
September 17, 2025 at 3:11 PM
We had some excellent talks by trainees and young investigators this morning! These speakers include (1/2):
- Sarah Grandinette, University of Pennsylvania
- Xiaoshu Pan, University of Florida
- Sarah Grandinette, University of Pennsylvania
- Xiaoshu Pan, University of Florida
Started off the day with welcome back remarks from Dr. Vivian Ota Wang.
September 17, 2025 at 1:13 PM
Started off the day with welcome back remarks from Dr. Vivian Ota Wang.
Ready for day 2 of the SCGE fall meeting!
September 17, 2025 at 12:33 PM
Ready for day 2 of the SCGE fall meeting!
Thank you to Drs. Liza-Marie Johnson and Akshay Sharma from St. Jude Children's Research Hospital for joining us for a great discussion on effective clinical trial design.
Check out this resource from their work in the Sickle Cell Disease Gene Therapy Working Group: myscdstory.org
Check out this resource from their work in the Sickle Cell Disease Gene Therapy Working Group: myscdstory.org
September 16, 2025 at 6:47 PM
Thank you to Drs. Liza-Marie Johnson and Akshay Sharma from St. Jude Children's Research Hospital for joining us for a great discussion on effective clinical trial design.
Check out this resource from their work in the Sickle Cell Disease Gene Therapy Working Group: myscdstory.org
Check out this resource from their work in the Sickle Cell Disease Gene Therapy Working Group: myscdstory.org
Group photo at the SCGE Fall 2025 meeting!
September 16, 2025 at 3:40 PM
Group photo at the SCGE Fall 2025 meeting!
Starting off the meeting strong with a great keynote presentation from Dr. Donald B. Kohn!
September 16, 2025 at 1:41 PM
Starting off the meeting strong with a great keynote presentation from Dr. Donald B. Kohn!
SCGE fall meeting is being set-up bright and early! Looking forward to seeing everyone soon!
September 16, 2025 at 11:44 AM
SCGE fall meeting is being set-up bright and early! Looking forward to seeing everyone soon!
Researchers - do you have any questions about gene therapy clinical trials that would help you in your research?
With our free Gene Therapy Clinical Trial Browser, you can get answers to these questions and more.
Check out the browser at scge.mcw.edu/platform/dat...
#Research #GeneTherapy 🧬🧪
With our free Gene Therapy Clinical Trial Browser, you can get answers to these questions and more.
Check out the browser at scge.mcw.edu/platform/dat...
#Research #GeneTherapy 🧬🧪
September 10, 2025 at 4:06 PM
Researchers - do you have any questions about gene therapy clinical trials that would help you in your research?
With our free Gene Therapy Clinical Trial Browser, you can get answers to these questions and more.
Check out the browser at scge.mcw.edu/platform/dat...
#Research #GeneTherapy 🧬🧪
With our free Gene Therapy Clinical Trial Browser, you can get answers to these questions and more.
Check out the browser at scge.mcw.edu/platform/dat...
#Research #GeneTherapy 🧬🧪
The SCGE Gene Therapy Clinical Trial Browser has many use cases for researchers, clinicians, patients, and more. Click through these images for examples of how different individuals could use the browser. 🧪🧬💻
To start your own search, go to scge.mcw.edu/platform/dat...
To start your own search, go to scge.mcw.edu/platform/dat...
September 3, 2025 at 9:46 PM
The SCGE Gene Therapy Clinical Trial Browser has many use cases for researchers, clinicians, patients, and more. Click through these images for examples of how different individuals could use the browser. 🧪🧬💻
To start your own search, go to scge.mcw.edu/platform/dat...
To start your own search, go to scge.mcw.edu/platform/dat...
In this project, SCGE researchers aim to optimize and evaluate their recently developed high-throughput extracellular vesicle (EV) loading platform with rigorous quality characterization on EVs and genome editor payload (1/2)
July 30, 2025 at 7:08 PM
In this project, SCGE researchers aim to optimize and evaluate their recently developed high-throughput extracellular vesicle (EV) loading platform with rigorous quality characterization on EVs and genome editor payload (1/2)
Want to read some interesting gene therapy research? Check out these publications from SCGE consortium research projects. 🧪🧬
Phase 1 Publications: scge.mcw.edu/publications/
Phase 2 Publications: scge.mcw.edu/phase-2-publ...
Phase 1 Publications: scge.mcw.edu/publications/
Phase 2 Publications: scge.mcw.edu/phase-2-publ...
June 9, 2025 at 2:17 PM
Want to read some interesting gene therapy research? Check out these publications from SCGE consortium research projects. 🧪🧬
Phase 1 Publications: scge.mcw.edu/publications/
Phase 2 Publications: scge.mcw.edu/phase-2-publ...
Phase 1 Publications: scge.mcw.edu/publications/
Phase 2 Publications: scge.mcw.edu/phase-2-publ...
In Phase 1, the SCGE program created a toolkit to promote the novel strategies and technologies from consortium projects.
In the toolkit, you can find info on:
- Genome editors ✂️
- Model systems 🐁
- Delivery systems 🚚
- Guides 🧬
- Vectors 🧪
- Protocols 📋
- And more! 🔬
scge.mcw.edu/toolkit/
In the toolkit, you can find info on:
- Genome editors ✂️
- Model systems 🐁
- Delivery systems 🚚
- Guides 🧬
- Vectors 🧪
- Protocols 📋
- And more! 🔬
scge.mcw.edu/toolkit/
May 30, 2025 at 1:42 PM
In Phase 1, the SCGE program created a toolkit to promote the novel strategies and technologies from consortium projects.
In the toolkit, you can find info on:
- Genome editors ✂️
- Model systems 🐁
- Delivery systems 🚚
- Guides 🧬
- Vectors 🧪
- Protocols 📋
- And more! 🔬
scge.mcw.edu/toolkit/
In the toolkit, you can find info on:
- Genome editors ✂️
- Model systems 🐁
- Delivery systems 🚚
- Guides 🧬
- Vectors 🧪
- Protocols 📋
- And more! 🔬
scge.mcw.edu/toolkit/
We've put together a (non-exhaustive) list of resources for researchers in the genome editing field. Check it out on our website: scge.mcw.edu/scge-resourc... 🧬🧪💻
May 19, 2025 at 2:11 PM
We've put together a (non-exhaustive) list of resources for researchers in the genome editing field. Check it out on our website: scge.mcw.edu/scge-resourc... 🧬🧪💻
Excited for another day of #ASGCT2025! Today you can hear from SCGE researchers Rebecca Ahrens-Nicklas, Fyodor Urnov, Allyson Berent, and a keynote presentation from Drew Weissman.🧪🧬
May 14, 2025 at 1:25 PM
Excited for another day of #ASGCT2025! Today you can hear from SCGE researchers Rebecca Ahrens-Nicklas, Fyodor Urnov, Allyson Berent, and a keynote presentation from Drew Weissman.🧪🧬