Casey Maguire
@casey-maguire-lab.bsky.social
In vivo gene delivery developer
Efficient Scaling up EV‐AAVs Production via Cellular Nanoporation for Familial Hypercholesterolaemia Therapy - Yan - 2025 - Journal of Extracellular Vesicles - Wiley Online Library isevjournals.onlinelibrary.wiley.com/doi/10.1002/...
November 16, 2025 at 12:13 AM
Efficient Scaling up EV‐AAVs Production via Cellular Nanoporation for Familial Hypercholesterolaemia Therapy - Yan - 2025 - Journal of Extracellular Vesicles - Wiley Online Library isevjournals.onlinelibrary.wiley.com/doi/10.1002/...
Reposted by Casey Maguire
New @nejm.org
One-shot gene therapy for a type of congenital deafness enabled some children to hear for the first time, and 3 of 12 kids achieved normal hearing
www.nejm.org/doi/full/10.1056/NEJMoa2400521
One-shot gene therapy for a type of congenital deafness enabled some children to hear for the first time, and 3 of 12 kids achieved normal hearing
www.nejm.org/doi/full/10.1056/NEJMoa2400521
DB-OTO Gene Therapy for Inherited Deafness | NEJM
Genetic deficiency of otoferlin, a protein critical to synaptic transmission by the sensory hair cells of the ear, causes congenital deafness. Medicines to treat the condition are lacking; children...
www.nejm.org
October 12, 2025 at 2:19 PM
New @nejm.org
One-shot gene therapy for a type of congenital deafness enabled some children to hear for the first time, and 3 of 12 kids achieved normal hearing
www.nejm.org/doi/full/10.1056/NEJMoa2400521
One-shot gene therapy for a type of congenital deafness enabled some children to hear for the first time, and 3 of 12 kids achieved normal hearing
www.nejm.org/doi/full/10.1056/NEJMoa2400521
Glad this is out- nice work by team Portugal! :)
October 12, 2025 at 3:20 AM
Glad this is out- nice work by team Portugal! :)
Thanks @bkleinstiver.bsky.social . Always enjoy the ride with you scientifically. And giving your cool editing systems a “smooth ride”! ;)
In vivo base editing via AAV9 or a smooth muscle cell tropic capsid AAV-PR (@casey-maguire-lab.bsky.social) substantially extended lifespan of treated MSMDS mice (+ key phenotypic changes). Notably, most AAV-ABE treated MSMDS mice died from bowel impaction, which should be avoidable in human.
September 18, 2025 at 2:29 AM
Thanks @bkleinstiver.bsky.social . Always enjoy the ride with you scientifically. And giving your cool editing systems a “smooth ride”! ;)
Reposted by Casey Maguire
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 Casey Maguire
🚨🚨🚨 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
July 29, 2025 at 5:32 PM
David Corey is one of the most brilliant scientists I’ve had the pleasure of working with. Cutting funding to scientists like him is going to hurt our country in so many ways including its standing in the world as well as economically. Not to mention the health of our citizens. All for what?
July 15, 2025 at 9:13 PM
David Corey is one of the most brilliant scientists I’ve had the pleasure of working with. Cutting funding to scientists like him is going to hurt our country in so many ways including its standing in the world as well as economically. Not to mention the health of our citizens. All for what?
Reposted by Casey Maguire
A troubling moment for the next generation of scientists.
UMass Chan Med School is rescinding dozens of PhD offers in biomedical sciences due to fears over federal research funding cuts.
www.nbcboston.com/news/local/u...
UMass Chan Med School is rescinding dozens of PhD offers in biomedical sciences due to fears over federal research funding cuts.
www.nbcboston.com/news/local/u...
UMass Chan rescinds admission for biomedical sciences PhD program amid funding concerns
UMass Chan Medical School is rescinding admission offers to candidates for its biomedical sciences PhD program over concerns about federal funding cuts.
www.nbcboston.com
March 13, 2025 at 5:13 PM
A troubling moment for the next generation of scientists.
UMass Chan Med School is rescinding dozens of PhD offers in biomedical sciences due to fears over federal research funding cuts.
www.nbcboston.com/news/local/u...
UMass Chan Med School is rescinding dozens of PhD offers in biomedical sciences due to fears over federal research funding cuts.
www.nbcboston.com/news/local/u...
Reposted by Casey Maguire
Broad Institute represented at #Standupforscience Boston
Emily Bramel from our research team with the proteosome poster 10/10
Emily Bramel from our research team with the proteosome poster 10/10
March 8, 2025 at 2:52 PM
Broad Institute represented at #Standupforscience Boston
Emily Bramel from our research team with the proteosome poster 10/10
Emily Bramel from our research team with the proteosome poster 10/10
Scientific innovation is a defining characteristic of our country. Don’t let that be a thing of the past! #StandUpforScience2025
March 7, 2025 at 8:43 PM
Scientific innovation is a defining characteristic of our country. Don’t let that be a thing of the past! #StandUpforScience2025
Reposted by Casey Maguire
The Trump administration continues its deluge of executive orders that directly affect science and research. Everything from cancer research to the distribution of medical information is affected. 🧪
www.iflscience.com/trump-impose...
www.iflscience.com/trump-impose...
Trump Imposes Immediate Restrictions On National Institutes Of Health With "Devastating" Impact
Everything from cancer research to the distribution of medical information is affected.
www.iflscience.com
January 24, 2025 at 12:01 PM
The Trump administration continues its deluge of executive orders that directly affect science and research. Everything from cancer research to the distribution of medical information is affected. 🧪
www.iflscience.com/trump-impose...
www.iflscience.com/trump-impose...
Our new publication characterizing the function of extracellular vesicle-associated AAV (EV-AAV) vectors is out!
We have developed the EV-AAV platform for several years, yet there is still much to learn about the unique AAV-based system.
We have developed the EV-AAV platform for several years, yet there is still much to learn about the unique AAV-based system.
January 20, 2025 at 5:40 AM
Our new publication characterizing the function of extracellular vesicle-associated AAV (EV-AAV) vectors is out!
We have developed the EV-AAV platform for several years, yet there is still much to learn about the unique AAV-based system.
We have developed the EV-AAV platform for several years, yet there is still much to learn about the unique AAV-based system.
Reposted by Casey Maguire
Have you heard the news? Addgene’s now offering AAV Packaged on Request! Read all about it in our latest blog post.
blog.addgene.org/aav...
blog.addgene.org/aav...
AAV Packaged on Request is Here!
Addgene's AAV Packaged on Request option has officially launched! Learn more about how this expansion of our helpful viral vectors service can help your research.
blog.addgene.org
January 14, 2025 at 3:00 PM
Have you heard the news? Addgene’s now offering AAV Packaged on Request! Read all about it in our latest blog post.
blog.addgene.org/aav...
blog.addgene.org/aav...
Reposted by Casey Maguire
On January 10th our viral vector service is expanding to include a packaged on request option! You will be able to choose a plasmid, select a preferred serotype, and submit a request — all in one place. Want to learn more? Sign up below for email updates.
info.addgene.org/pac...
info.addgene.org/pac...
January 3, 2025 at 5:56 PM
On January 10th our viral vector service is expanding to include a packaged on request option! You will be able to choose a plasmid, select a preferred serotype, and submit a request — all in one place. Want to learn more? Sign up below for email updates.
info.addgene.org/pac...
info.addgene.org/pac...
Reposted by Casey Maguire
We're expanding our viral vectors service to include AAV Packaged on Request — and the ordering process will look a little different! Here's what you need to know:
blog.addgene.org/add...
blog.addgene.org/add...
December 16, 2024 at 7:05 PM
We're expanding our viral vectors service to include AAV Packaged on Request — and the ordering process will look a little different! Here's what you need to know:
blog.addgene.org/add...
blog.addgene.org/add...
Please see our new publication, Metabolic engineering improves transduction efficiency and downstream vector isolation by altering the lipid composition of extracellular vesicle-enclosed AAV by Espinoza et al.
Metabolic engineering improves transduction efficiency and downstream vector isolation by altering the lipid composition of extracellular vesicle-enclosed AAV
Adeno-associated viruses (AAV) are promising vectors for gene therapy due to their efficacy in vivo. However, there is room for improvement to address…
www.sciencedirect.com
December 12, 2024 at 2:50 PM
Please see our new publication, Metabolic engineering improves transduction efficiency and downstream vector isolation by altering the lipid composition of extracellular vesicle-enclosed AAV by Espinoza et al.
Reposted by Casey Maguire
Check out our Clinical Perspective on the recent clinical trials of gene therapy for treating deafness, and on its remaining limitations with delivery issues and low spectrum of cell types targeted. Together with Landegger/Reisinger/Hage/Grimm/Cederroth labs.
www.cell.com/molecular-th...
www.cell.com/molecular-th...
The rise of cochlear gene therapy
Landegger and colleagues discuss several recent clinical trials that have demonstrated
gene therapy’s safety and effectiveness in treating hearing loss. While many questions
remain, ongoing research a...
www.cell.com
November 19, 2024 at 7:04 AM
Check out our Clinical Perspective on the recent clinical trials of gene therapy for treating deafness, and on its remaining limitations with delivery issues and low spectrum of cell types targeted. Together with Landegger/Reisinger/Hage/Grimm/Cederroth labs.
www.cell.com/molecular-th...
www.cell.com/molecular-th...