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
In this video of the SCGE Meet the Expert Webinar Series, panelists discuss different economic and reimbursement perspectives of gene editing therapies. Watch the full video at: www.youtube.com/watch?v=Oo41...
Meet the Expert: Shyam Patel, Kevin Strauss, Manar Zaghlula
YouTube video by SCGE Outreach
youtube.com
Collagen type I, a key component of the extracellular matrix, underpins cellular organization and regeneration but remains challenging to process into scalable, reproducible, and biologically faithful 3D materials. (1/4)
High-throughput Generation of Collagen Microbeads for Extracellular Vesicle Production and Therapeutic Delivery
Collagen type I, a key component of the extracellular matrix, underpins cellular organization and regeneration but remains challenging to process into scalable, reproducible, and biologically faithful...
pmc.ncbi.nlm.nih.gov
In our latest video in the SCGE "Meet the Expert" series, panelists share their experiences with rare disease and provide patient and caregiver perspectives on gene therapy for hearing and vision loss. Watch the full video at www.youtube.com/watch?v=z_WQ...
Meet the Expert: Jessica Chaikof, Rachel Chaikof, and Stacey Ashlund
YouTube video by SCGE Outreach
youtube.com
TnpB is a diverse family of RNA-guided endonucleases associated with prokaryotic transposons. Because of their small size and putative evolutionary relationship to CRISPR–Cas12, TnpB enzymes hold great potential for genome editing. However, most TnpBs lack robust gene-editing activity. (1/4)
Engineered TnpB genome editors for plants and human cells identified by ribonucleoprotein mutational scanning - Nature Biotechnology
TnpB endonucleases are engineered for improved genome editing.
nature.com
In this edition of the SCGE Spotlight, we're covering: - Prime editing using LNPs - Gene editing in human kidney organoids - A toolkit for IND applications - Interviews with SCGE researchers www.linkedin.com/pulse/scge-s...
SCGE Spotlight #4
In this edition of the SCGE Spotlight, we’re sharing new publications, a collection of resources to help fill out an IND submission, and more SCGE researcher interviews. Let’s get started.
linkedin.com
Interactions between genes or cis-regulatory elements (CREs) underlie many biological processes. High-throughput CRISPR screens have allowed researchers to assess the impact of activation or repression of gene and regulatory elements on many phenotypes. (1/3) www.nature.com/articles/s41...
dHyperCas12a enables multiplexed CRISPRi screens - Nature Communications
CRISPR/Cas9 screens have identified genetic contributions to many phenotypes. However, studying combinations of genes or regulatory elements remains challenging. Here, the authors use CRISPR/Cas12a to...
nature.com
In this interview, Marsha Michie, PhD and Megan Allyse, PhD, discuss a range of ethics topics related to gene therapy, from adapting patient educational materials to the ethical considerations around somatic cell in utero gene editing therapies. Watch the interview: www.youtube.com/watch?v=r_vt...
Meet the Researcher: Marsha Michie and Megan Allyse
YouTube video by SCGE Outreach
youtube.com
Extracellular vesicles (EVs) have emerged as promising nanocarriers for cancer therapy given their high biocompatibility and low immunogenicity. However, their clinical utility remains limited by challenges such as off-target, premature drug release, and rapid clearance. (1/5)
Engineering stimuli-responsive extracellular vesicles for enhanced anticancer therapeutics
Extracellular vesicles (EVs), so called nano-sized vesicles shedding out from cells, have emerged as promising nanocarriers for cancer therapy given their high biocompatibility and low immunogenicity....
pmc.ncbi.nlm.nih.gov
We're thrilled to announce the Center for Therapeutic Genetics (CTG) — a new collaboration between JAX, @broadinstitute.org, and @bostonchildrens.bsky.social to develop genetic medicines for rare and ultra-rare diseases. 🔬🧪🧬 go.jax.org/Center-Therapeutic-Genetics
Broad Institute, Boston Children’s Hospital, and The Jackson Laboratory launch the Center for Therapeutic Genetics
A new collaboration to develop precision medicines for those with rare diseases, using infrastructure and repeatable practices that will be shared with others.
go.jax.org
In this "Meet the Researcher” interview, hear from Christopher Baehr, PhD, as he discusses innovative approaches for improving patient access to gene therapies and shares his ideas and hopes for the gene therapy field in the next decade. @chrisbaehr.bsky.social www.youtube.com/watch?v=yS6l...
Meet the Researcher: Chris Baehr
YouTube video by SCGE Outreach
youtube.com
Synthetic biology offers control over cellular and tissue functions. As it moves beyond microbes into humans, synthetic biology enables precise control over gene expression, cell fate, and tissue organization across heart, lung, blood, and sleep systems. (1/4)
Integrating synthetic biology to understand and engineer the heart, lung, blood, and sleep systems
Synthetic biology offers control over cellular and tissue functions. As it moves beyond microbes into humans, synthetic biology enables precise contro…
sciencedirect.com
In this interview, Greg Mohl, PhD highlights the challenges of delivering CRISPR therapies to the brain and discusses how him and the research team try to balance designing the most effective therapy with the time pressure to get therapies to patients. www.youtube.com/watch?v=QGQd...
Meet the Researcher: Greg Mohl
YouTube video by SCGE Outreach
youtube.com
The U.S. Food and Drug Administration (FDA)'s platform technology designation program aims to streamline the development and approval process for advanced therapy medicinal products and is anticipated to be beneficial for the development of gene therapies for rare and ultra-rare diseases. (1/3)
In this interview, Jennifer Panagoulias provides her perspective on some current regulatory frameworks, and shares practical advice for academic labs preparing their first IND application for a gene therapy. Watch the full interview at: www.youtube.com/watch?v=53WP...
Meet the Researcher: Jennifer Panagoulias
YouTube video by SCGE Outreach
youtube.com
We're excited to share the third edition of the SCGE Spotlight! Read the newsletter at www.linkedin.com/pulse/scge-s...
SCGE Spotlight #3: June 2026
Hello from the third edition of the SCGE Spotlight! We’ve got two more publications, some free educational materials geared towards the general public, and SCGE researcher interviews. Let’s get starte...
linkedin.com
In this study, SCGE researchers isolated a library of natural capsids from human tissues and identified two variants that conferred strong transduction of retinal tissues following intravitreal injections into mice and non-human primates. (1/3)
sciencedirect.com
Industry News: Beam Therapeutics Announces Clearance of Investigational New Drug Application for BEAM-304 for the Treatment of Phenylketonuria (PKU) by the United States (U.S.) Food and Drug Administration investors.beamtx.com/news-release...
investors.beamtx.com
In this "Meet the Researcher Interview", hear from Karen Clark, PhD, as she highlights the free resources available on the SCGE platform. www.youtube.com/watch?v=O2gj...
Meet the Researcher: Karen Clark
YouTube video by SCGE Outreach
youtube.com
The efficacy of photothermal therapy (PTT) of cancer critically depends on widespread nanoparticle (NP) distribution and retention within the tumor. (1/4)
MPI‐Guided Photothermal Therapy of Prostate Cancer Using Stem Cell Delivery of Magnetotheranostic Nanoflowers
Tumor-tropic human mesenchymal stem cells (hMSCs) were used as delivery vehicles for magnetotheranostic gold–iron oxide nanoflowers. Magnetic particle imaging of the iron component demonstrated wides....
advanced.onlinelibrary.wiley.com
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: www.youtube.com/watch?v=0HTR...
Meet the Researcher: Xiaoshu Pan
YouTube video by SCGE Outreach
youtube.com
CRISPR-based genome editing therapeutics are entering the clinic, offering transformative potential but also presenting potential risks. Preclinical-to-clinical toolkits are needed to assess the safety and efficacy of these new therapies and accelerate progress. (1/3) www.nature.com/articles/s41...
Monitoring biological effects of somatic cell genome editing - Nature Reviews Genetics
CRISPR-based genome editing therapeutics are entering the clinic, but in vitro and in vivo tools are needed to assess their safety and efficacy. The authors review complementary technologies to monito...
nature.com
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 different kinds of expertise needed to build her research project. www.youtube.com/watch?v=cmor...
Meet the Researcher: Mei He
YouTube video by SCGE Outreach
youtube.com
Autosomal recessive polycystic kidney disease (ARPKD) leads to severe renal cysts and progressive kidney dysfunction, with no approved treatments. The absence of such cystic phenotypes in Pkhd1-/- mice underscores the need for novel models that better recapitulate the human disease. (1/5)
Deciphering the Impact of RAC1‐SPTAN1 in ARPKD Cystogenesis Using Multifaceted Models
Distal/connecting tubules expressing SLC8A1 have been suggested as a potential origin of ARPKD cysts. SPTAN1 has been identified as a key molecule in ARPKD cyst formation. Restoring SPTAN1 in PKHD1−/...
advanced.onlinelibrary.wiley.com
The second edition of the SCGE Spotlight is out now! Check out the newsletter at: www.linkedin.com/pulse/scge-s...
SCGE Spotlight #2: May 2026
Welcome to the second edition of the SCGE Spotlight! We’ve got some interesting publications, another free resource, and a few researcher wins to highlight. Let’s get started.
linkedin.com
Emerging therapies in sickle cell disease (SCD) aim to restore healthy red blood cell (RBC) function, but they often yield heterogeneous cellular responses. There are no proven techniques to evaluate restored rheological functionality and heterogeneity in these RBCs. (1/3)
Microfluidic capillary transit velocity as a functional measure for sickle cell disease and in vitro-derived red blood cells
Emerging therapies in sickle cell disease (SCD) aim to restore healthy red blood cell (RBC) function, but they often yield heterogeneous cellular responses. There are no proven techniques to evaluate ...
pubs.rsc.org
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/dat... #GeneTherapy #Regulatory #FreeResource
Natural killer (NK) cells have been engineered to express chimeric antigen receptors (CARs) to enhance their cytotoxic capabilities through CAR-mediated activation, a strategy that has yielded promising advancements in cancer treatment in recent pre-clinical and clinical trials. (1/3)
Frontiers | Perspective: IL-15 cytokine-armored NK cells as ready-to-use immunotherapy for diverse malignancies: therapeutic potential and toxicity risks
Natural killer (NK) cells have been engineered to express chimeric antigen receptors (CARs) to enhance their cytotoxic capabilities through CAR-mediated acti...
frontiersin.org
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)
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)
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)
