The Somatic Cell Genome Editing Consortium is funded by the @NIH_CommonFund. Retweets ≠ endorsements. Visit the SCGE Platform scge.mcw.edu/platform/
Joined September 2019
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644 Photos and videos
CRISPR-based gene editing therapies are entering the clinic, offering transformative potential but also presenting potential risks. Preclinical-to-clinical toolkits are needed to assess safety and efficacy of these new therapies and accelerate progress.
nature.com/articles/s41576-0…
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In this SCGE "Meet the Researcher" interview, hear from Dr. Xiaoshu Pan as she highlights the benefits of extracellular vesicles (EVs) as a delivery method for gene therapies, and discusses the next milestones in her SCGE project.
Watch the interview: youtube.com/watch?v=0HTRPl2X…
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Dr. Pan is a Research Assistant Professor at the University of Florida College of Pharmacy, and is part of the SCGE project titled "Evaluation of High-throughput Extracellular Vesicle Loading Platform for Therapeutic Genome Editing INDs".
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Using a multifaceted approach, SCGE researchers identified reduced levels of SPTAN1, a cytoskeletal spectrin protein, as a key regulator of RAC1 activation and cystic pathology in autosomal recessive polycystic kidney disease (ARPKD).
advanced.onlinelibrary.wiley…
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In this SCGE researcher interview, Dr. Mei He highlights how her teams' high-throughput extracellular vesicle loading platform improves the delivery of genome editors and discusses the breadth of expertise needed to build her research project. @UF_HE_LAB
youtube.com/watch?v=cmoriIih…
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Dr. He is an Associate Professor at the University of Florida College of Pharmacy, and is the PI on the SCGE project titled "Evaluation of High-throughput Extracellular Vesicle Loading Platform for Therapeutic Genome Editing INDs".
scge.mcw.edu/phase-2-technol…
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Somatic Cell Genome Editing (SCGE) Outreach retweeted
Jun 2
Today, the FDA published a draft guidance that when finalized will outline how sponsors can use publicly available information and established platform knowledge to streamline the development of cell and gene therapy products. fda.gov/news-events/press-an…
ALT Grey graphic with text stating "FDA issues draft guidance to streamline development of cell and gene therapy products." The background features multiple faded instances of the words "DRAFT GUIDANCE." The FDA logo is in the bottom right corner.
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Somatic Cell Genome Editing (SCGE) Outreach retweeted
Jun 1
On Thursday June 4, 2026, from 11 am – 12 PM ET, the Office of Therapeutic Products will host a virtual town hall with experts from OTP’s Office of Review Management & Regulatory Review answering questions regarding Biologic License Application readiness. fda.gov/vaccines-blood-biolo…
ALT A graphic for an OTP Town Hall event by the FDA. The topic is "Best Practices for Preparing BLA Submissions for Cell and Gene Therapy Products." Scheduled for Thursday, June 4, 2026, from 11:00 a.m. to 12:00 p.m. ET. The event will be streamed on YouTube, and a recording will be available. The FDA logo is present. No registration is required.
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Here, SCGE researchers present a biomimetic microcapillary network, high-speed imaging, and computational algorithms to analyze red blood cell capillary velocity profiles of the entire sample population at single-cell resolution. #PublicationFriday
pmc.ncbi.nlm.nih.gov/article…
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Somatic Cell Genome Editing (SCGE) Outreach retweeted
May 27
Congratulations to our research team with Zachary F. Greenberg Brian Iván Molina Díaz Samantha Ali for receiving the NIH Replication Prize Track I Award🎉
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The second edition of the SCGE Spotlight is out now! Check out the newsletter at:
linkedin.com/pulse/scge-spot…
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SCGE researchers speculate that the use of humanized mice will allow for engraftment of tumor and alleviate cytokine armored CAR-NK toxicity, thereby allowing for effective assessment of CAR-NK efficacy in the absence of toxicity. #PublicationFriday
frontiersin.org/journals/imm…
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Are you part of a research team that is submitting (or planning to submit) an Investigational New Drug (IND) application for a novel gene therapy?
View our free IND application resources at scge.mcw.edu/platform/data/i…
#GeneTherapy #Regulatory #FreeResource
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Somatic Cell Genome Editing (SCGE) Outreach retweeted
May 18
Atamyo Therapeutics Reports Promising Early Results from the First Patients Treated with ATA-200 Gene Therapy in its LGMD-R5 Clinical Trial - For More Information Visit shorturl.at/fKQqN @AtamyoTX #LGMD #LGMD_R5 #Limb_Girdle #Muscular_Dystrophy #Rare_diseases
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Somatic Cell Genome Editing (SCGE) Outreach retweeted
May 15
One-Time Gene Therapy Shows Potential to Improve Bone Health in Adults With Gaucher Disease - For More Information Visit shorturl.at/bWtVd @SpurTherapeutx #Gaucher_Disease #Rare_Diseases #Gaucher
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SCGE researchers recently published an article on peptide-enabled ribonucleoprotein delivery for CRISPR engineering (PERC), a new approach for ex vivo genome editing of primary human cells.
#PublicationFriday
nature.com/articles/s41596-0…
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Somatic Cell Genome Editing (SCGE) Outreach retweeted
May 14
Don’t miss today’s George Stamatoyannopoulos Memorial Symposium featuring three visionary keynote speakers:
🔹Paula Cannon, PhD: Designing what nature can’t: Cell and gene therapy for HIV
🔹Erik Sontheimer, PhD: CRISPR, from bacterial immunity to genome editing therapeutics
🔹Beverly Davidson, PhD: Outstanding Achievement Award Recipient
We'll also be celebrating the recipients of the George Stamatoyannopoulos Mentorship Award, the Excellence in Research Award, and the Mavis Agbandje-McKenna Scholarship and Fund. See you there!
Session details: pag.virtual-meeting.org/US/a…
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Somatic Cell Genome Editing (SCGE) Outreach retweeted
Thursday May 14th:
- Beverly Davidson, Outstanding Achievement Award Winner
- Samantha Maragh, "NIST Genome Editing Consortium expanded and extended to develop genome editing control strategies, tools, samples, and standards" (8/9)
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Somatic Cell Genome Editing (SCGE) Outreach retweeted
Thursday May 14th:
- Beno Freedman, "Using human iPS cell-derived organoids and CRISPR gene editing in kidney diseases"
- Alan Flake, "Artificial womb to support fetal development and manipulations" (7/9)
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Somatic Cell Genome Editing (SCGE) Outreach retweeted
Thursday May 14th:
- Danilo Pellin, "Metagenomic integration analysis of hematopoietic stem cell following lentiviral gene transfer"
- Chris Baehr, "Chemically modified CRISPR enzymes for multi-organ genome editing in vivo" (6/9)
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