Kiran Musunuru
@kiranmusunuru.bsky.social
Cardiologist, geneticist, and gene editor. Author of #TheCRISPRGeneration and #GenomeEditingAPracticalGuide
She Was Born Without an Immune System. Gene Therapy Saved Her Life.
www.nytimes.com/2025/11/27/w...
www.nytimes.com/2025/11/27/w...
She Was Born Without an Immune System. Gene Therapy Saved Her Life.
www.nytimes.com
November 27, 2025 at 5:02 PM
She Was Born Without an Immune System. Gene Therapy Saved Her Life.
www.nytimes.com/2025/11/27/w...
www.nytimes.com/2025/11/27/w...
Reposted by Kiran Musunuru
🚨Big News!🚨
Our keynote lineup for the ASHG Genetic Diagnosis & Rare Disease Virtual Symposium is here!
Dec 2: @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social
Dec. 3: Helene Cederroth & Eric Klee
You won't want to miss their insights shaping rare disease genetics: bit.ly/3WVd6l2 #ASHG
Our keynote lineup for the ASHG Genetic Diagnosis & Rare Disease Virtual Symposium is here!
Dec 2: @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social
Dec. 3: Helene Cederroth & Eric Klee
You won't want to miss their insights shaping rare disease genetics: bit.ly/3WVd6l2 #ASHG
November 21, 2025 at 4:22 PM
🚨Big News!🚨
Our keynote lineup for the ASHG Genetic Diagnosis & Rare Disease Virtual Symposium is here!
Dec 2: @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social
Dec. 3: Helene Cederroth & Eric Klee
You won't want to miss their insights shaping rare disease genetics: bit.ly/3WVd6l2 #ASHG
Our keynote lineup for the ASHG Genetic Diagnosis & Rare Disease Virtual Symposium is here!
Dec 2: @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social
Dec. 3: Helene Cederroth & Eric Klee
You won't want to miss their insights shaping rare disease genetics: bit.ly/3WVd6l2 #ASHG
Reposted by Kiran Musunuru
Recent news reports highlighting commercial efforts to normalize heritable human genome editing (HHGE) underscore the urgent need for sustained vigilance, ethical clarity, and enforceable global governance. Read more: https://bit.ly/4r8iIGt
Global Scientific Consortium Reaffirms Position on Human Heritable…
Recent news reports highlighting commercial efforts to normalize heritable human genome editing (HHGE) underscore the urgent need for sustained vigilance,…
www.asgct.org
November 18, 2025 at 10:52 PM
Recent news reports highlighting commercial efforts to normalize heritable human genome editing (HHGE) underscore the urgent need for sustained vigilance, ethical clarity, and enforceable global governance. Read more: https://bit.ly/4r8iIGt
Reposted by Kiran Musunuru
New 🧬✂️ pre-print! We show that paired prime editing can efficiently generate large deletions — even >1 Mb — with high precision and at scale. We use this to perform the first pooled prime deletion screen across the human genome.
🔗 biorxiv.org/content/10.1...
A short thread (by Juliane Weller)👇
🔗 biorxiv.org/content/10.1...
A short thread (by Juliane Weller)👇
Generating long deletions across the genome with pooled paired prime editing screens
Engineered deletions are a powerful probe for studying genome architecture, function, and regulation. Yet, the lack of effective methods to create them in large numbers and at multi-kilobase scale has...
biorxiv.org
November 5, 2025 at 2:17 PM
New 🧬✂️ pre-print! We show that paired prime editing can efficiently generate large deletions — even >1 Mb — with high precision and at scale. We use this to perform the first pooled prime deletion screen across the human genome.
🔗 biorxiv.org/content/10.1...
A short thread (by Juliane Weller)👇
🔗 biorxiv.org/content/10.1...
A short thread (by Juliane Weller)👇
Reposted by Kiran Musunuru
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 Kiran Musunuru
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 Kiran Musunuru
Can personalized treatment become the standard of care? Just published in @ajhgnews.bsky.social, @kiranmusunuru.bsky.social, MD, PhD, and @ahrensnicklas.bsky.social, MD, PhD, explore their vision for the future of interventional genetics: www.cell.com/ajhg/fulltex... #ASHG #HumanGenetics
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 3:57 PM
Can personalized treatment become the standard of care? Just published in @ajhgnews.bsky.social, @kiranmusunuru.bsky.social, MD, PhD, and @ahrensnicklas.bsky.social, MD, PhD, explore their vision for the future of interventional genetics: www.cell.com/ajhg/fulltex... #ASHG #HumanGenetics
Reposted by Kiran Musunuru
Super cool and important new work in gene dysregulation by HMGN1 as a mechanism for heart defects #CHD in Down Syndrome. From my @gladstoneinst.bsky.social colleagues led by @sanjeevranade.bsky.social www.nature.com/articles/s41...
Myocardial reprogramming by HMGN1 underlies heart defects in trisomy 21 - Nature
Studies using human pluripotent stem cells and a mouse model of Down syndrome identify HMGN1 as a key contributor to congenital heart defects in individuals with Down syndrome.
www.nature.com
October 22, 2025 at 4:26 PM
Super cool and important new work in gene dysregulation by HMGN1 as a mechanism for heart defects #CHD in Down Syndrome. From my @gladstoneinst.bsky.social colleagues led by @sanjeevranade.bsky.social www.nature.com/articles/s41...
Reposted by Kiran Musunuru
The astonishing science of synthetic embryology and our profile of it's most ambitious praticioner, Jacob Hanna
www.technologyreview.com/2025/10/21/1...
www.technologyreview.com/2025/10/21/1...
The astonishing embryo models of Jacob Hanna
Scientists are creating the beginnings of bodies without sperm or eggs. How far should they be allowed to go?
www.technologyreview.com
October 21, 2025 at 6:06 PM
The astonishing science of synthetic embryology and our profile of it's most ambitious praticioner, Jacob Hanna
www.technologyreview.com/2025/10/21/1...
www.technologyreview.com/2025/10/21/1...
Reposted by Kiran Musunuru
“We must keep going with this ability to sample genomes, not just in the convenient places we started out with.” - former Director of the National Institutes of Health, Francis Collins, MD, PhD, during his recognition speech at #ASHG25
October 14, 2025 at 9:11 PM
“We must keep going with this ability to sample genomes, not just in the convenient places we started out with.” - former Director of the National Institutes of Health, Francis Collins, MD, PhD, during his recognition speech at #ASHG25
Reposted by Kiran Musunuru
🤗 Out now @naturemedicine.bsky.social results of our genomic NBS study BabyScreen+ 👶🧬
👉 www.nature.com/articles/s41...
1,000 babies
WGS using existing cards
600+ conditions
13 day TAT
16 diagnoses (vs 1 in std NBS)
High clinical impact
High parental acceptability
👉 www.nature.com/articles/s41...
1,000 babies
WGS using existing cards
600+ conditions
13 day TAT
16 diagnoses (vs 1 in std NBS)
High clinical impact
High parental acceptability
October 9, 2025 at 8:03 PM
🤗 Out now @naturemedicine.bsky.social results of our genomic NBS study BabyScreen+ 👶🧬
👉 www.nature.com/articles/s41...
1,000 babies
WGS using existing cards
600+ conditions
13 day TAT
16 diagnoses (vs 1 in std NBS)
High clinical impact
High parental acceptability
👉 www.nature.com/articles/s41...
1,000 babies
WGS using existing cards
600+ conditions
13 day TAT
16 diagnoses (vs 1 in std NBS)
High clinical impact
High parental acceptability
Parents of children with rare diseases ask: How long until our CRISPR miracle? www.statnews.com/2025/10/09/c... via @statnews.com
Parents of children with rare diseases ask: How long until our CRISPR miracle?
How to better manage expectations around gene editing drugs for families of children with rare diseases.
www.statnews.com
October 9, 2025 at 8:54 AM
Parents of children with rare diseases ask: How long until our CRISPR miracle? www.statnews.com/2025/10/09/c... via @statnews.com
These Patients Got the Cure. Then It Went Away.
Gene therapies for rare diseases are frequently developed then discarded by drug companies because they can’t afford to produce the treatment for more patients.
www.nytimes.com/2025/09/22/h...
Gene therapies for rare diseases are frequently developed then discarded by drug companies because they can’t afford to produce the treatment for more patients.
www.nytimes.com/2025/09/22/h...
These Patients Got the Cure. Then It Went Away.
www.nytimes.com
September 22, 2025 at 5:24 PM
These Patients Got the Cure. Then It Went Away.
Gene therapies for rare diseases are frequently developed then discarded by drug companies because they can’t afford to produce the treatment for more patients.
www.nytimes.com/2025/09/22/h...
Gene therapies for rare diseases are frequently developed then discarded by drug companies because they can’t afford to produce the treatment for more patients.
www.nytimes.com/2025/09/22/h...
Reposted by Kiran Musunuru
Optimization of a bespoke base editor to treat a severe pediatric vascular disease! 🫀🧬
Our manuscript describes:
1️⃣ Engineering a target-specific BE🧬
2⃣ A *must avoid* bystander edit that occurs with WT SpCas9 BEs! 🙅♂️
3⃣ Extension of lifespan after in vivo editing! 🐁✅
www.nature.com/articles/s41...
Our manuscript describes:
1️⃣ Engineering a target-specific BE🧬
2⃣ A *must avoid* bystander edit that occurs with WT SpCas9 BEs! 🙅♂️
3⃣ Extension of lifespan after in vivo editing! 🐁✅
www.nature.com/articles/s41...
Treatment of a severe vascular disease using a bespoke CRISPR–Cas9 base editor in mice - Nature Biomedical Engineering
Engineering a mutant-specific customized base editor precisely corrects a mutation while minimizing bystander edits, leading to substantial phenotypic recovery in mouse models of multisystemic smooth ...
www.nature.com
September 12, 2025 at 2:00 AM
Optimization of a bespoke base editor to treat a severe pediatric vascular disease! 🫀🧬
Our manuscript describes:
1️⃣ Engineering a target-specific BE🧬
2⃣ A *must avoid* bystander edit that occurs with WT SpCas9 BEs! 🙅♂️
3⃣ Extension of lifespan after in vivo editing! 🐁✅
www.nature.com/articles/s41...
Our manuscript describes:
1️⃣ Engineering a target-specific BE🧬
2⃣ A *must avoid* bystander edit that occurs with WT SpCas9 BEs! 🙅♂️
3⃣ Extension of lifespan after in vivo editing! 🐁✅
www.nature.com/articles/s41...
Reposted by Kiran Musunuru
🚨🚨🚨 Finally out!!, Our study describing the development and deployment of a bespoke CRISPR–Cas9 base editor for palliation of a severe monogenic vasculopathy by genome editing is published today in Nature Biomedical Engineering
🧵 to follow
Here is a free link: rdcu.be/eFAEz
🧵 to follow
Here is a free link: rdcu.be/eFAEz
Treatment of a severe vascular disease using a bespoke CRISPR–Cas9 base editor in mice
Nature Biomedical Engineering - Engineering a mutant-specific customized base editor precisely corrects a mutation while minimizing bystander edits, leading to substantial phenotypic recovery in...
rdcu.be
September 11, 2025 at 2:41 PM
🚨🚨🚨 Finally out!!, Our study describing the development and deployment of a bespoke CRISPR–Cas9 base editor for palliation of a severe monogenic vasculopathy by genome editing is published today in Nature Biomedical Engineering
🧵 to follow
Here is a free link: rdcu.be/eFAEz
🧵 to follow
Here is a free link: rdcu.be/eFAEz
In a Rock on Mars, NASA Sees ‘Clearest Sign of Life’ So Far
www.nytimes.com
September 10, 2025 at 6:24 PM
Are A.I. Tools Making Doctors Worse at Their Jobs? www.nytimes.com/2025/08/28/w...
Are A.I. Tools Making Doctors Worse at Their Jobs?
www.nytimes.com
September 1, 2025 at 11:10 PM
Are A.I. Tools Making Doctors Worse at Their Jobs? www.nytimes.com/2025/08/28/w...
Reposted by Kiran Musunuru
Our latest research is out today on @medrxivpreprint.bsky.social:
www.medrxiv.org/content/10.1...
Saturation genome editing of BRCA1 across cell types accurately resolves cancer risk.
Led by the amazing Phoebe Dace. This one’s packed full of data, so check out the paper. Quick highlights… 🧵 1/n
www.medrxiv.org/content/10.1...
Saturation genome editing of BRCA1 across cell types accurately resolves cancer risk.
Led by the amazing Phoebe Dace. This one’s packed full of data, so check out the paper. Quick highlights… 🧵 1/n
Saturation genome editing of BRCA1 across cell types accurately resolves cancer risk
Germline pathogenic BRCA1 variants predispose women to breast and ovarian cancer. Despite accumulation of functional evidence for variants in BRCA1 , over half of reported single-nucleotide variants (...
www.medrxiv.org
August 18, 2025 at 7:33 AM
Our latest research is out today on @medrxivpreprint.bsky.social:
www.medrxiv.org/content/10.1...
Saturation genome editing of BRCA1 across cell types accurately resolves cancer risk.
Led by the amazing Phoebe Dace. This one’s packed full of data, so check out the paper. Quick highlights… 🧵 1/n
www.medrxiv.org/content/10.1...
Saturation genome editing of BRCA1 across cell types accurately resolves cancer risk.
Led by the amazing Phoebe Dace. This one’s packed full of data, so check out the paper. Quick highlights… 🧵 1/n
Once a Death Sentence, This Heart Condition Is Finally Treatable www.nytimes.com/2025/08/04/w...
Once a Death Sentence, This Heart Condition Is Finally Treatable
www.nytimes.com
August 4, 2025 at 9:50 PM
Once a Death Sentence, This Heart Condition Is Finally Treatable www.nytimes.com/2025/08/04/w...
Reposted by Kiran Musunuru
Congrats to @ahrensnicklas.bsky.social for being named Rad Girl of the Year at the 2025 Rad Awards! Dr. Ahrens-Nicklas was nominated for her groundbreaking work in personalized gene therapy with baby KJ.
Learn more about the 2025 Rad Award winners at thephiladelphiacitizen.org/2025-rad-awa...
Learn more about the 2025 Rad Award winners at thephiladelphiacitizen.org/2025-rad-awa...
Celebrating Rad Women at the 2025 Rad Awards
On July 30, Philadelphia turned out to joyfully celebrate the women who are making our lives much, much better
thephiladelphiacitizen.org
August 4, 2025 at 2:17 PM
Congrats to @ahrensnicklas.bsky.social for being named Rad Girl of the Year at the 2025 Rad Awards! Dr. Ahrens-Nicklas was nominated for her groundbreaking work in personalized gene therapy with baby KJ.
Learn more about the 2025 Rad Award winners at thephiladelphiacitizen.org/2025-rad-awa...
Learn more about the 2025 Rad Award winners at thephiladelphiacitizen.org/2025-rad-awa...
Reposted by Kiran Musunuru
🧬 NOW OPEN: Registration for Breakthroughs in Targeted In Vivo Gene Editing!
On Nov. 20-21, dive into cutting-edge solutions for targeted delivery of #geneediting technology in vivo in San Diego and virtually. www.asgct.org/events/break...
On Nov. 20-21, dive into cutting-edge solutions for targeted delivery of #geneediting technology in vivo in San Diego and virtually. www.asgct.org/events/break...
July 28, 2025 at 4:52 PM
🧬 NOW OPEN: Registration for Breakthroughs in Targeted In Vivo Gene Editing!
On Nov. 20-21, dive into cutting-edge solutions for targeted delivery of #geneediting technology in vivo in San Diego and virtually. www.asgct.org/events/break...
On Nov. 20-21, dive into cutting-edge solutions for targeted delivery of #geneediting technology in vivo in San Diego and virtually. www.asgct.org/events/break...
Reposted by Kiran Musunuru
This is a new image from JWST.
The six-pointed features are stars in the Milky Way.
Everything else is a galaxy.
Everything else is a galaxy.
The six-pointed features are stars in the Milky Way.
Everything else is a galaxy.
Everything else is a galaxy.
June 30, 2025 at 4:15 PM
This is a new image from JWST.
The six-pointed features are stars in the Milky Way.
Everything else is a galaxy.
Everything else is a galaxy.
The six-pointed features are stars in the Milky Way.
Everything else is a galaxy.
Everything else is a galaxy.
Reposted by Kiran Musunuru
🚨🚨🚨Big news for development of in vivo CAR T cells: AbbVie (ABBV) to Acquire Capstan Therapeutics for $2.1 Billion @abbvie @capstantx #TCell #tcellrx #immunotherapy www.streetinsider.com/Corporate+Ne...
AbbVie (ABBV) to Acquire Capstan Therapeutics for $2.1 Billion
AbbVie (NYSE: ABBV) and Capstan Therapeutics, Inc. ("Capstan"), a clinical-stage biotechnology company dedicated to advancing in vivo engineering of cells through RNA delivery using tLNPs, today...
www.streetinsider.com
June 30, 2025 at 12:37 PM
🚨🚨🚨Big news for development of in vivo CAR T cells: AbbVie (ABBV) to Acquire Capstan Therapeutics for $2.1 Billion @abbvie @capstantx #TCell #tcellrx #immunotherapy www.streetinsider.com/Corporate+Ne...
Reposted by Kiran Musunuru
Look at those two spiral galaxies. Two Milky Ways. Two hundreds of billions of stars and planets. Two homes.
*slams fists onto table* DAMMIT the universe is SO COOL.
*slams fists onto table* DAMMIT the universe is SO COOL.
June 23, 2025 at 5:27 AM
Look at those two spiral galaxies. Two Milky Ways. Two hundreds of billions of stars and planets. Two homes.
*slams fists onto table* DAMMIT the universe is SO COOL.
*slams fists onto table* DAMMIT the universe is SO COOL.
People With Severe Diabetes Are Cured in Small Trial of New Drug www.nytimes.com/2025/06/20/h...
People With Severe Diabetes Are Cured in Small Trial of New Drug
www.nytimes.com
June 21, 2025 at 12:51 AM
People With Severe Diabetes Are Cured in Small Trial of New Drug www.nytimes.com/2025/06/20/h...