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
Reposted by SCGE
We’d love to see you next week at Breakthroughs in Targeted In Vivo Gene Editing – in San Diego or online! You’ll hear new research from CGT leaders, including these featured speakers and many more: asgct.org/breakthroughs
November 13, 2025 at 4:07 PM
We’d love to see you next week at Breakthroughs in Targeted In Vivo Gene Editing – in San Diego or online! You’ll hear new research from CGT leaders, including these featured speakers and many more: asgct.org/breakthroughs
Reposted by SCGE
Check out the Q3 Landscape Report for the latest in the therapeutics pipeline, clinical targets, developer progress, and more! 🧬 Read the full report: https://www.asgct.org/news-publications/landscape-report
November 12, 2025 at 7:06 PM
Check out the Q3 Landscape Report for the latest in the therapeutics pipeline, clinical targets, developer progress, and more! 🧬 Read the full report: https://www.asgct.org/news-publications/landscape-report
In this video of the SCGE Meet the Expert series, panelists use their legal and U.S. Food and Drug Administration (FDA) expertise to provide practical guidance to rare disease researchers in preclinical stages of development.
Watch the full video at www.youtube.com/watch?v=0U1x...
Watch the full video at www.youtube.com/watch?v=0U1x...
Meet the Expert: James Valentine and Richard Lewis
YouTube video by SCGE Outreach
www.youtube.com
November 12, 2025 at 2:36 PM
In this video of the SCGE Meet the Expert series, panelists use their legal and U.S. Food and Drug Administration (FDA) expertise to provide practical guidance to rare disease researchers in preclinical stages of development.
Watch the full video at www.youtube.com/watch?v=0U1x...
Watch the full video at www.youtube.com/watch?v=0U1x...
CRISPR-Cas12a enzymes' dependence on a 5'-TTTV-3' protospacer adjacent motif (PAM) next to DNA target sequences restricts Cas12a's gene targeting capability to only ∼1% of a typical genome. (1/3)
academic.oup.com/nar/article/...
academic.oup.com/nar/article/...
November 11, 2025 at 3:20 PM
CRISPR-Cas12a enzymes' dependence on a 5'-TTTV-3' protospacer adjacent motif (PAM) next to DNA target sequences restricts Cas12a's gene targeting capability to only ∼1% of a typical genome. (1/3)
academic.oup.com/nar/article/...
academic.oup.com/nar/article/...
Reposted by SCGE
Coming up *tomorrow* November 6th — join us to hear about the latest in #CRISPR for human health from IGI's Brad Ringeisen, Fyodor Urnov, and Carlotta Ronda as part of @ucberkeleyofficial.bsky.social's Basic Science Lights the Way lecture series! Register here: https://basicscience.berkeley.edu/
November 5, 2025 at 5:00 PM
Coming up *tomorrow* November 6th — join us to hear about the latest in #CRISPR for human health from IGI's Brad Ringeisen, Fyodor Urnov, and Carlotta Ronda as part of @ucberkeleyofficial.bsky.social's Basic Science Lights the Way lecture series! Register here: https://basicscience.berkeley.edu/
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...
Reposted by SCGE
📢 AVAILABLE NOW: Breakthroughs in Targeted In Vivo Gene Editing abstracts!
View and download the collection of original research on our website, and don't miss your chance to dive deeper Nov. 20-21 in San Diego and virtually. https://bit.ly/47R1ndv
View and download the collection of original research on our website, and don't miss your chance to dive deeper Nov. 20-21 in San Diego and virtually. https://bit.ly/47R1ndv
Breakthroughs in Targeted In Vivo Gene Editing
Dedicated to cutting-edge solutions for targeted delivery, this event is your chance to engage with pioneering research, overcome tissue-specific…
www.asgct.org
November 3, 2025 at 11:15 PM
📢 AVAILABLE NOW: Breakthroughs in Targeted In Vivo Gene Editing abstracts!
View and download the collection of original research on our website, and don't miss your chance to dive deeper Nov. 20-21 in San Diego and virtually. https://bit.ly/47R1ndv
View and download the collection of original research on our website, and don't miss your chance to dive deeper Nov. 20-21 in San Diego and virtually. https://bit.ly/47R1ndv
The SCGE site is undergoing scheduled maintenance on 11/3 from approximately 11:00am-2:00pm CT. We are sorry for any inconvenience.
November 3, 2025 at 5:10 PM
The SCGE site is undergoing scheduled maintenance on 11/3 from approximately 11:00am-2:00pm CT. We are sorry for any inconvenience.
Reposted by SCGE
Read more about today's paper (tinyurl.com/23c5vzd9) from @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social that discusses regulatory innovation in the pathway toward interventional genetics!
Personalized gene editing helped one baby: can it be rolled out widely?
In a world first, a bespoke gene-editing therapy benefitted one child. Now reseachers plan to launch a clinical trial of the approach.
www.nature.com
October 31, 2025 at 6:47 PM
Read more about today's paper (tinyurl.com/23c5vzd9) from @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social that discusses regulatory innovation in the pathway toward interventional genetics!
Reposted by SCGE
In this article, SCGE researchers Rebecca Ahrens-Nicklas and Kiran Musunuru discuss the next steps towards creating personalized gene editing platforms.
Read the full paper at: www.cell.com/ajhg/fulltex...
@ahrensnicklas.bsky.social @kiranmusunuru.bsky.social
Read the full paper at: www.cell.com/ajhg/fulltex...
@ahrensnicklas.bsky.social @kiranmusunuru.bsky.social
How to create personalized gene editing platforms: Next steps toward interventional genetics
How do we go from a single individual receiving a personalized gene-editing therapy
to a future of “interventional genetics” in which such therapies are the standard
of care? First and foremost: regul...
www.cell.com
October 31, 2025 at 1:13 PM
In this article, SCGE researchers Rebecca Ahrens-Nicklas and Kiran Musunuru discuss the next steps towards creating personalized gene editing platforms.
Read the full paper at: www.cell.com/ajhg/fulltex...
@ahrensnicklas.bsky.social @kiranmusunuru.bsky.social
Read the full paper at: www.cell.com/ajhg/fulltex...
@ahrensnicklas.bsky.social @kiranmusunuru.bsky.social
Reposted by SCGE
🚨Online NOW!!
📄How to create personalized gene editing platforms: Next steps toward interventional genetics
🧑🤝🧑 @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social
📄How to create personalized gene editing platforms: Next steps toward interventional genetics
🧑🤝🧑 @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social
How to create personalized gene editing platforms: Next steps toward interventional genetics
How do we go from a single individual receiving a personalized gene-editing therapy
to a future of “interventional genetics” in which such therapies are the standard
of care? First and foremost: regul...
www.cell.com
October 31, 2025 at 1:03 PM
🚨Online NOW!!
📄How to create personalized gene editing platforms: Next steps toward interventional genetics
🧑🤝🧑 @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social
📄How to create personalized gene editing platforms: Next steps toward interventional genetics
🧑🤝🧑 @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social
Reposted by SCGE
Tune in soon to hear more from @kiranmusunuru.bsky.social & @ahrensnicklas.bsky.social !!
The scientists behind Baby KJ’s life-saving CRISPR therapy have a plan to help more patients. Join me and @drewsnews.bsky.social at 9:45 for a conversation with UPenn's Kiran Musunuru and CHOP's Rebecca Ahrens-Nicklas to learn more! @endpts.com endpoints.news/post-hoc-liv...
Watch at 9:45 a.m.: Meet the scientists behind Baby KJ’s life-saving CRISPR therapy
U Penn's Musunuru and CHOP's Ahrens-Nicklas discuss plans to expand Baby KJ's custom CRISPR therapy for rare genetic diseases into an FDA-approved treatment.
endpoints.news
October 31, 2025 at 1:34 PM
Tune in soon to hear more from @kiranmusunuru.bsky.social & @ahrensnicklas.bsky.social !!
In this article, SCGE researchers Rebecca Ahrens-Nicklas and Kiran Musunuru discuss the next steps towards creating personalized gene editing platforms.
Read the full paper at: www.cell.com/ajhg/fulltex...
@ahrensnicklas.bsky.social @kiranmusunuru.bsky.social
Read the full paper at: www.cell.com/ajhg/fulltex...
@ahrensnicklas.bsky.social @kiranmusunuru.bsky.social
How to create personalized gene editing platforms: Next steps toward interventional genetics
How do we go from a single individual receiving a personalized gene-editing therapy
to a future of “interventional genetics” in which such therapies are the standard
of care? First and foremost: regul...
www.cell.com
October 31, 2025 at 1:13 PM
In this article, SCGE researchers Rebecca Ahrens-Nicklas and Kiran Musunuru discuss the next steps towards creating personalized gene editing platforms.
Read the full paper at: www.cell.com/ajhg/fulltex...
@ahrensnicklas.bsky.social @kiranmusunuru.bsky.social
Read the full paper at: www.cell.com/ajhg/fulltex...
@ahrensnicklas.bsky.social @kiranmusunuru.bsky.social
The difficulty of delivering genes to the kidney has limited the translation of genetic medicines, particularly for the more than 10% of the global population with chronic kidney disease. (1/3)
www.nature.com/articles/s41...
www.nature.com/articles/s41...
Evolving adeno-associated viruses for gene transfer to the kidney via cross-species cycling of capsid libraries - Nature Biomedical Engineering
Variants of adeno-associated viruses displaying robust and widespread transduction in murine, porcine and non-human-primate kidneys, and in human kidney organoids, can be obtained via the cross-specie...
www.nature.com
October 28, 2025 at 1:42 PM
The difficulty of delivering genes to the kidney has limited the translation of genetic medicines, particularly for the more than 10% of the global population with chronic kidney disease. (1/3)
www.nature.com/articles/s41...
www.nature.com/articles/s41...
Reposted by SCGE
In this video of the Meet the Expert Series, Deanna Portero and Stefano Benvenuti discuss alternative business models for non-commercially viable diseases.
Check out this thoughtful discussion at: www.youtube.com/watch?v=1WeB...
#RareDisease #OrphanDrug #Biotech
Check out this thoughtful discussion at: www.youtube.com/watch?v=1WeB...
#RareDisease #OrphanDrug #Biotech
Meet the Expert: Deanna Portero and Stefano Benvenuti
YouTube video by SCGE Outreach
www.youtube.com
October 22, 2025 at 1:44 PM
In this video of the Meet the Expert Series, Deanna Portero and Stefano Benvenuti discuss alternative business models for non-commercially viable diseases.
Check out this thoughtful discussion at: www.youtube.com/watch?v=1WeB...
#RareDisease #OrphanDrug #Biotech
Check out this thoughtful discussion at: www.youtube.com/watch?v=1WeB...
#RareDisease #OrphanDrug #Biotech
In this video of the Meet the Expert Series, Deanna Portero and Stefano Benvenuti discuss alternative business models for non-commercially viable diseases.
Check out this thoughtful discussion at: www.youtube.com/watch?v=1WeB...
#RareDisease #OrphanDrug #Biotech
Check out this thoughtful discussion at: www.youtube.com/watch?v=1WeB...
#RareDisease #OrphanDrug #Biotech
Meet the Expert: Deanna Portero and Stefano Benvenuti
YouTube video by SCGE Outreach
www.youtube.com
October 22, 2025 at 1:44 PM
In this video of the Meet the Expert Series, Deanna Portero and Stefano Benvenuti discuss alternative business models for non-commercially viable diseases.
Check out this thoughtful discussion at: www.youtube.com/watch?v=1WeB...
#RareDisease #OrphanDrug #Biotech
Check out this thoughtful discussion at: www.youtube.com/watch?v=1WeB...
#RareDisease #OrphanDrug #Biotech
The apolipoprotein E (APOE) ε4 allele is widely recognized as the strongest genetic risk factor for late-onset Alzheimer's disease. 🧵(1/4)
alz-journals.onlinelibrary.wiley.com/doi/10.1002/...
alz-journals.onlinelibrary.wiley.com/doi/10.1002/...
Focused ultrasound‐mediated APOE4 knockdown in mouse brain
INTRODUCTION The apolipoprotein E (APOE) ε4 allele is widely recognized as the strongest genetic risk factor for late-onset Alzheimer's disease. Therapeutic strategies to reduce apoE4 expression in ...
alz-journals.onlinelibrary.wiley.com
October 21, 2025 at 5:45 PM
The apolipoprotein E (APOE) ε4 allele is widely recognized as the strongest genetic risk factor for late-onset Alzheimer's disease. 🧵(1/4)
alz-journals.onlinelibrary.wiley.com/doi/10.1002/...
alz-journals.onlinelibrary.wiley.com/doi/10.1002/...
In the latest video of the Meet the Expert Series, panelists Nick Buss, Ilan Irony, and Allen Callaway discuss non-clinical study design and regulatory insights for seamless transitions. 🧬🧪
Watch the video at www.youtube.com/watch?v=WrFu...
#Research #GeneTherapy #Regulatory #Webinar
Watch the video at www.youtube.com/watch?v=WrFu...
#Research #GeneTherapy #Regulatory #Webinar
Meet the Expert: Nick Buss, Ilan Irony, Allen Callaway
YouTube video by SCGE Outreach
www.youtube.com
October 15, 2025 at 1:20 PM
In the latest video of the Meet the Expert Series, panelists Nick Buss, Ilan Irony, and Allen Callaway discuss non-clinical study design and regulatory insights for seamless transitions. 🧬🧪
Watch the video at www.youtube.com/watch?v=WrFu...
#Research #GeneTherapy #Regulatory #Webinar
Watch the video at www.youtube.com/watch?v=WrFu...
#Research #GeneTherapy #Regulatory #Webinar
Hear from SCGE researcher Kris Saha in this new ASGCT podcast episode!
www.asgct.org/education-ca...
@sahakris.bsky.social @asgct.bsky.social
www.asgct.org/education-ca...
@sahakris.bsky.social @asgct.bsky.social
A Molecular Glue Degrader to Control CRISPR with Drs.… | ASGCT
On this episode of The Molecular Therapy Podcast, listen to Drs. Krishanu Saha and Namita Khajanchi discuss their article that was recently published in…
www.asgct.org
October 13, 2025 at 2:14 PM
Hear from SCGE researcher Kris Saha in this new ASGCT podcast episode!
www.asgct.org/education-ca...
@sahakris.bsky.social @asgct.bsky.social
www.asgct.org/education-ca...
@sahakris.bsky.social @asgct.bsky.social
Comparative studies to optimize homology-dependent repair (HDR) parameters for introducing base pair changes using short-read deep sequencing have been successful, but similar analysis for insertions is challenging due to read-length constraints. (1/5) 🧵🧪🧬
journals.biologists.com/dev/article/...
journals.biologists.com/dev/article/...
Identifying optimal conditions for precise knock-in of exogenous DNA into the zebrafish genome
Summary: Quantitative side-by-side comparison of commonly used homology templates and CRISPR nucleases allows identification of optimal conditions for germline transmission of precise insertions in ze...
journals.biologists.com
October 7, 2025 at 2:05 PM
Comparative studies to optimize homology-dependent repair (HDR) parameters for introducing base pair changes using short-read deep sequencing have been successful, but similar analysis for insertions is challenging due to read-length constraints. (1/5) 🧵🧪🧬
journals.biologists.com/dev/article/...
journals.biologists.com/dev/article/...
A team of SCGE researchers assessed the spillover effect within a field of view containing multiple hot spots, a scenario frequently encountered in vivo after systemic administration of a magnetic tracer. 🧵(1/3) 🧬🧪
www.nature.com/articles/s44...
www.nature.com/articles/s44...
Spillover can limit accurate signal quantification in MPI - npj Imaging
npj Imaging - Spillover can limit accurate signal quantification in MPI
www.nature.com
September 30, 2025 at 5:01 PM
A team of SCGE researchers assessed the spillover effect within a field of view containing multiple hot spots, a scenario frequently encountered in vivo after systemic administration of a magnetic tracer. 🧵(1/3) 🧬🧪
www.nature.com/articles/s44...
www.nature.com/articles/s44...
Some human genes contain highly repetitive sequences of bases that can grow in length uncontrollably. These repeat sequences cause cells to either produce bulky, toxic proteins or stop making essential proteins altogether. 🧵(1/5)
commonfund.nih.gov/editing/high...
commonfund.nih.gov/editing/high...
Gene Editing Technology Disrupts Mutation that Causes Huntington’s Disease and Friedrich’s Ataxia | NIH Common Fund
Genetic information in the human body is stored as code inside DNA. This code is written using four different building blocks, called bases. Different combinations of these bases encode different mess...
commonfund.nih.gov
September 24, 2025 at 8:24 PM
Some human genes contain highly repetitive sequences of bases that can grow in length uncontrollably. These repeat sequences cause cells to either produce bulky, toxic proteins or stop making essential proteins altogether. 🧵(1/5)
commonfund.nih.gov/editing/high...
commonfund.nih.gov/editing/high...
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.
That's a wrap on the Fall 2025 SCGE meeting. Thanks to everyone who participated in-person and virtually!
Check out a brief recap of the meeting at the link below.
scge.mcw.edu/2025/09/17/s...
Check out a brief recap of the meeting at the link below.
scge.mcw.edu/2025/09/17/s...
SCGE Fall 2025 Meeting
Check out this photo recap of the SCGE Consortium in-person meeting taking place in September 2025 in Arlington, VA.
scge.mcw.edu
September 17, 2025 at 6:52 PM
That's a wrap on the Fall 2025 SCGE meeting. Thanks to everyone who participated in-person and virtually!
Check out a brief recap of the meeting at the link below.
scge.mcw.edu/2025/09/17/s...
Check out a brief recap of the meeting at the link below.
scge.mcw.edu/2025/09/17/s...