@bohlen.bsky.social
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For the oculomotor nerds out there⦠weāre excited to share our paired papers on precerebellar circuitry! These two studies look at how internuclear pathways talk to the cerebellum and what that means for eye movement control. šš§
#neuroskyence
#science
3 days ago
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reposted by
Cell Reports Medicine
5 days ago
Online now: Complete neutralizing antibody evasion by serodivergent non-mammalian AAVs enables gene therapy redosing
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Complete neutralizing antibody evasion by serodivergent non-mammalian AAVs enables gene therapy redosing
Loeb et al. develop an immune-evasive gene therapy vector derived from Muscovy duck, termed AAV.div3A. Rational engineering yields a liver-detargeted variant with enhanced transduction in the heart and diaphragm. AAV.div3A fully evades pre-existing antibodies and vector-induced immunity, enabling effective gene therapy redosing in a Pompe disease model.
http://dlvr.it/TPWLZh
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reposted by
Waggoner Lab
7 days ago
Complete neutralizing antibody evasion by serodivergent non-mammalian AAVs enables gene therapy redosing
@cp-cellrepmed.bsky.social
www.cell.com/cell-reports...
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reposted by
Nonia Pariente
7 days ago
Happy to see the field advance in leaps and bounds. Great news for terrible diseases! When I postdocād developing lentivectors for gene therapy it was already used for severe combined immunodeficiency, but other applications seemed quite far away and the field was in the post-hype depression. š§Ŗ
add a skeleton here at some point
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reposted by
UCL Discovery
6 days ago
Open Access UCL Research: Gene therapy for Parkinson's disease: trials and technical advances
discovery.ucl.ac.uk/id/eprint/10...
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Gene therapy for Parkinson's disease: trials and technical advances - UCL Discovery
UCL Discovery is UCL's open access repository, showcasing and providing access to UCL research outputs from all UCL disciplines.
https://discovery.ucl.ac.uk/id/eprint/10210619/
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Primates are absolutely critical for developing safe and effective gene therapies
add a skeleton here at some point
7 days ago
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reposted by
7 days ago
Signal or noise? > Viral Vectors-based Gene Therapy for Non-human Primates Market - openPR.com >> Comment below!
#strategy
#competitiveintelligence
#marketing
#healthcare
#biotech
#pharmaceutical
#pharma
#competitivemarketing
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Viral Vectors-based Gene Therapy for Non-human Primates Market - openPR.com
Viral Vectors-based Gene Therapy for Non-human Primates MarketĀ Ā openPR.com
http://dlvr.it/TPTLvb
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reposted by
22 days ago
A top FDA regulator has announced that the agency plans to relax its strict rules for gene therapy development in an effort to fast-track gene therapies and boost investment in experimental treatments.
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FDA clears way for faster personalized gene editing therapy
The FDA plans to unveil a new approval process for custom gene-editing, a move designed to unleash a wave of industry investment. Read on
https://financialpost.com/technology/fda-gene-editing-therapy
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reposted by
Michele A. Basso
27 days ago
Fear, monkeys, and institutionalĀ courage When news broke of an explosion inside Harvardās neurobiologyĀ buildingĀ early Saturday morning, every scientist who works with monkeys felt itāan involuntary jolt, a spike of cortisol, the silent thought:Ā what if it had been us? Neither animals nor peopleā¦
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Fear, monkeys, and institutionalĀ courage
When news broke of an explosion inside Harvardās neurobiologyĀ buildingĀ early Saturday morning, every scientist who works with monkeys felt itāan involuntary jolt, a spike of cortisol, the silent thought:Ā what if it had been us? Neither animals nor people were hurt, thankfully. ItĀ doesnātĀ appear to have been an attack. But it didnāt matter. For those of us who work with monkeys, the fear is always near the surface.
https://micheleabasso7.wordpress.com/2025/11/06/fear-monkeys-and-institutional-courage/
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reposted by
Michael Okun
26 days ago
We desperately need new research tools to move the ball forward in Parkinson's. How about seeing dopamine in a new light? Enter 'far-red sensors' that unlock multiplex brain signals. Could a far-red dopamine sensor open the door to simultaneously watching multiple brain chemicals 'in action.'
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reposted by
bioRxiv Neuroscience
about 1 month ago
Single-cell, multi-region profiling of the macaque brain across the lifespan
https://www.biorxiv.org/content/10.1101/2025.10.31.685880v1
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reposted by
The Transmitter
about 1 month ago
Dutch lawmakers have approved the phase-out of primate research at one of Europeās biggest facilities. Neuroscientists are worried. By Lauren Schenkman
#neuroskyence
www.thetransmitter.org/animal-model...
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Nonhuman primate research to lose federal funding at major European facility
The Dutch Senate has ordered the Biomedical Primate Research Centre in the Netherlands to shift its funding away from primate experiments by 2030.
https://www.thetransmitter.org/animal-models/nonhuman-primate-research-to-lose-federal-funding-at-major-european-facility/?utm_source=bluesky&utm_medium=org-social&utm_campaign=20251030-news-nonhuman-primate-lose-federal-funding-european
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reposted by
BYTESEU
about 1 month ago
AAV Quantification: Towards Faster, More Scalable Analysis
https://www.byteseu.com/1496260/
Adeno-associated virus (AAV) is widely used in gene therapy as a vector to deliver genetic material into patient cells due to its specificity and low immunogenicity. Accurate AAV quantification and ā¦
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AAV Quantification: Towards Faster, More Scalable Analysis - Bytes Europe
Adeno-associated virus (AAV) is widely used in gene therapy as a vector to deliver genetic material into patient cells due to its specificity and low
https://www.byteseu.com/1496260/
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reposted by
Stock Master
about 2 months ago
Teenager who received Elevidys, Sarepta's Duchenne gene therapy, dies - STAT Sarepta Therapeutics said that a young man died after receiving Elevidys, its gene therapy for Duchenne muscular dystrophy
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reposted by
PessoaBrain
about 2 months ago
š š”ššØš„š¢ššš¢šš¢ššššš š£šš„š¦š£ššš§šš©š š¢š” š§šš š£š„ššš„š¢š”š§šš šš¢š„š§šš« By Mars and Passingham "Understanding anthropoid foraging challenges may thus contribute to our understanding of human cognition" Going to the top of the reading list!
doi.org/10.1016/j.ne...
#neuroskyence
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reposted by
about 2 months ago
Signal or noise? > AviadoBio Looks to China for Eye-Based Gene Therapy in $413M UgeneX Pact - BioSpace >> Comment below!
#strategy
#competitiveintelligence
#marketing
#competitivemarketing
#pharma
#biotech
#pharmaceutical
#healthcare
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AviadoBio Looks to China for Eye-Based Gene Therapy in $413M UgeneX Pact - BioSpace
AviadoBio Looks to China for Eye-Based Gene Therapy in $413M UgeneX PactĀ Ā BioSpace
http://dlvr.it/TNbWVn
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reposted by
Ryan Cross
about 2 months ago
#ESGCT2025
: A woman who was previously ineligible for
@genethonfr.bsky.social
's CriglerāNajjar syndrome gene therapy due to pre-existing immunity to AAV8 got the treatment after Hansa Bio's antibody-chopping drug imlifidase cleared the way. Read more in
@endpts.com
:
endpoints.news/new-study-su...
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Person previously ineligible for gene therapy granted treatment after antibody-chopping drug clears the way
Study shows Hansa's Idefirix helps deliver Genethon's AAV8 gene therapy GNT003 in previously ineligible Crigler-Najjar patient by clearing antibodies, reports Do Cao at ESGCT.
https://endpoints.news/new-study-suggests-patients-with-anti-aav-antibodies-may-still-receive-gene-therapy/
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reposted by
about 2 months ago
Signal or noise? > FDA awards RMAT designation to Dravet syndrome gene therapy - Dravet Syndrome News >> Comment below!
#strategy
#competitiveintelligence
#marketing
#pharmaceutical
#healthcare
#biotech
#pharma
#competitivemarketing
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FDA awards RMAT designation to Dravet syndrome gene therapy - Dravet Syndrome News
FDA awards RMAT designation to Dravet syndrome gene therapyĀ Ā Dravet Syndrome News
http://dlvr.it/TNXVHn
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reposted by
Towers Lab
2 months ago
New preprint from our lab, led by our postdocs Dara Annett and Kate Morling in collaboration with Selwood Lab! We developed a modified cyclosporine, BG147, which enhances lentivector gene therapy transduction, ex vivo in HSPC and in vivo in mouse photoreceptors, by degrading IFITM3. Check it out!
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A modified cyclosporine enhances lentivector transduction ex vivo and in vivo by degrading IFITM3
Intrinsic innate immune barriers have evolved to suppress viral infection and can reduce effective gene delivery in gene therapy. We have developed BG147, a novel cyclosporine A analogue, optimised vi...
https://www.biorxiv.org/content/10.1101/2025.08.26.669098v1
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reposted by
Pluchino lab - Regenerative Neuroimmunology
2 months ago
Oligodendrocyte-targeted adeno-associated virus gene therapy for Canavan disease in children: a phase 1/2 trial
www.nature.com/articles/s41...
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Oligodendrocyte-targeted adeno-associated virus gene therapy for Canavan disease in children: a phase 1/2 trial - Nature Medicine
Interim trial results of MYR-101, a novel recombinant vector with selective tropism for oligodendrocytes, support its tolerability and indicate improved myelination and reduced cerebrospinal fluid N-acetyl-aspartic acid concentrations in children with Canavan disease.
https://www.nature.com/articles/s41591-025-03919-w#rightslink
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reposted by
Maria Carrillo
3 months ago
Proud to share the Alzheimerās Association invested $112.2M in research activities over the last year ā our largest single-year commitment. From gene therapy to
#BrainHealth
to risk reduction to AI, weāre funding bold boundary-pushing ideas.
alz.org/research
#ENDALZ
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Alzheimer's & Dementia Research | Alzheimer's Association
Alzheimer's and dementia research ā find the latest update on research funding, grants, clinical trials and global research news.
https://alz.org/research
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reposted by
Ellen Eldridge
3 months ago
Childrenās Healthcare of Atlanta recently performed Georgiaās first commercial gene therapy transplant for a genetic disease requiring lifelong blood transfusions.
www.gpb.org/news/2025/09...
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This teenager no longer needs lifelong blood transfusions thanks to gene transplant therapy
Childrenās Healthcare of Atlanta recently performed Georgiaās first commercial gene therapy transplant for a genetic disease requiring lifelong blood transfusions.
https://www.gpb.org/news/2025/09/15/teenager-no-longer-needs-lifelong-blood-transfusions-thanks-gene-transplant-therapy
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reposted by
Melayna
3 months ago
First Child Dosed in Gene Therapy Trial Dies.
www.medpagetoday.com/neurology/ge...
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First Child Dosed in Gene Therapy Trial Dies
Investigational agent aimed to treat rare gene-related encephalopathy
https://www.medpagetoday.com/neurology/generalneurology/117425
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reposted by
barry
3 months ago
Feeling a bit tearful at the moment. A gene therapy for RRP, the airway disease I've had for 25 years now, has been developed and is now approved. For those of us who've had anything between 50-500 throat operations, this is an absolute life changer!
biotechhealthx.com/biotech-news...
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Precigen (PGEN) Makes History: FDA Approves First-Ever Therapy Targeting Root Cause of RRP
Precigenās PAPZIMEOS therapy is transforming the lives of adults with recurrent respiratory papillomatosis, eliminating endless surgeries.
https://biotechhealthx.com/biotech-news/precigen-pgen-makes-history-fda-approves-first-ever-therapy-targeting-root-cause-of-rrp/
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Woof
www.geneonline.com/biotech-layo...
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Biotech Layoffs in 2025 Reflect Funding Gaps, Failed Trials, and Industry Pivots - GeneOnline News
If you feel like youāve seen nonstop headlines about job layoffs this year, youāre not imagining things. A report from a global outplacement and executive coaching firm shows that through July, U.S. e...
https://www.geneonline.com/biotech-layoffs-in-2025-reflect-funding-gaps-failed-trials-and-pharma-pivots/
3 months ago
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reposted by
4 months ago
NCBN Trending: Gene therapy startup Kriya Therapeutics gets $313M
https://zpr.io/V27wP4Znjfxt
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reposted by
4 months ago
Thoughts on this? >> Research Spotlight: Organoids Could Make Gene Therapy Trials Safer By Identifying Hidden Risks Early On - massgeneralbrigham.org >> Comment below!
#strategy
#competitiveintelligence
#marketing
#pharma
#competitivemarketing
#biotech
#healthcare
#pharmaceutical
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Research Spotlight: Organoids Could Make Gene Therapy Trials Safer By Identifying Hidden Risks Early On - massgeneralbrigham.org
Research Spotlight: Organoids Could Make Gene Therapy Trials Safer By Identifying Hidden Risks Early OnĀ Ā massgeneralbrigham.org
http://dlvr.it/TMX53y
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reposted by
The New York Times
4 months ago
Dr. Vinay Prasad, who led the FDAās vaccines and gene therapy division before resigning under pressure two weeks ago, is returning to the agency, a Department of Health and Human Services spokesman confirmed Saturday.
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Ousted F.D.A. Vaccine Chief Returns to Agency
Dr. Vinay Prasadās rehiring was an unusual instance of a federal official targeted by the right-wing activist Laura Loomer being brought back into the Trump administration.
https://nyti.ms/4lh0AWH
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reposted by
PaPaCarl
4 months ago
Story about my autistic grandson, Paxton Purdy, who has a terminal genetic mutation called CLCN6. Now heās at real risk of losing grants for his life saving gene therapy and for what? So billionaires can have more yachts! I just want to hang my head and cry!
www.cbsnews.com/losangeles/n...
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Family raises money for son's gene therapy and clinical trial to treat rare genetic mutation
Paxton Purdy's parents said he has an ultra-rare neurodegenerative genetic mutation. They hope they can raise enough money to cure it and help children in similar situations.
https://www.cbsnews.com/losangeles/news/family-raises-money-for-sons-gene-therapy-and-clinical-trial-to-treat-rare-genetic-mutation/
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reposted by
4 months ago
Thoughts on this? >> AAV in Gene Therapy Pipeline 2025: Mechanism of Action, Route - openPR.com >> Comment below!
#strategy
#competitiveintelligence
#marketing
#pharma
#biotech
#competitivemarketing
#healthcare
#pharmaceutical
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AAV in Gene Therapy Pipeline 2025: Mechanism of Action, Route - openPR.com
AAV in Gene Therapy Pipeline 2025: Mechanism of Action, RouteĀ Ā openPR.com
http://dlvr.it/TMKFKW
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reposted by
4 months ago
Thoughts on this? >> Imlifidase enables gene therapy in Duchenne patients with AAV antibodies - European Biotechnology Magazine >> Comment below!
#strategy
#competitiveintelligence
#marketing
#pharma
#pharmaceutical
#competitivemarketing
#biotech
#healthcare
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Imlifidase enables gene therapy in Duchenne patients with AAV antibodies - European Biotechnology Magazine
Imlifidase enables gene therapy in Duchenne patients with AAV antibodiesĀ Ā European Biotechnology Magazine
http://dlvr.it/TMHndG
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reposted by
4 months ago
Thoughts on this? >> AAV2-BDNF Gene Therapy Restores FDG-PET Activity in Entorhinal Cortex, Phase 1 Data Show - NeurologyLive >> Comment below!
#strategy
#competitiveintelligence
#marketing
#competitivemarketing
#pharma
#healthcare
#biotech
#pharmaceutical
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AAV2-BDNF Gene Therapy Restores FDG-PET Activity in Entorhinal Cortex, Phase 1 Data Show - NeurologyLive
AAV2-BDNF Gene Therapy Restores FDG-PET Activity in Entorhinal Cortex, Phase 1 Data ShowĀ Ā NeurologyLive
http://dlvr.it/TMDGCp
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reposted by
4 months ago
Study Highlights Need for Standardized Measurement Methods in Gene Therapy
https://www.newsbeep.com/uk/28256/
Some gene therapy treatments use modified adeno-associated viruses (AAVs) like the one shown here to deliver therapeutic geneticā¦
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Study Highlights Need for Standardized Measurement Methods in Gene Therapy - United Kingdom News Beep
Some gene therapy treatments use modified adeno-associated viruses (AAVs) like the one shown here to deliver therapeutic genetic material into a patientās
https://www.newsbeep.com/uk/28256/
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reposted by
Patrick C Miller
4 months ago
The deadly saga of the controversial gene therapy Elevidys
buff.ly/tJneVRW
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The deadly saga of the controversial gene therapy Elevidys
The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.
https://buff.ly/AdrwRMH
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A good day to be inside doing science.
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4 months ago
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reposted by
StockTitan
4 months ago
#CRL
Charles River Joins Ellyās Team to Drive Rare Disease Gene Therapy Development
https://www.stocktitan.net/news/CRL/charles-river-joins-elly-s-team-to-drive-rare-disease-gene-therapy-f4ycpcggc1od.html
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Charles River Joins Ellyās Team to Drive Rare Disease Gene Therapy Development
Charles River Laboratories (NYSE: CRL) has entered into a strategic CDMO agreement with Elly's Team, a parent-led foundation focused on developing treatments for NEDAMSS, an ultra-rare genetic disorder. The collaboration leverages Charles River's Cell and Gene Therapy Accelerator Program to manufacture critical materials for Phase I clinical trials.The partnership has already achieved significant success, with Elly becoming the first patient to receive IRF2BPL gene replacement therapy on April 3, 2025. The treatment received FDA approval in March 2025, just 13 months after diagnosis. Elly's Team utilized Charles River's eXpDNA⢠platform and AAV Rep/Cap plasmids, reducing manufacturing efforts by up to 66 percent.NEDAMSS, caused by IRF2BPL gene mutations, affects fewer than 150 cases worldwide and impacts motor skills, speech, eating, eyesight, and can cause seizures.
https://www.stocktitan.net/news/CRL/charles-river-joins-elly-s-team-to-drive-rare-disease-gene-therapy-f4ycpcggc1od.html
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reposted by
Ophthopedia
5 months ago
Ophthopedia Update: FDA fast tracks intravitreal gene therapy for geographic atrophy: Editorās note: This is a developing news story. Please check back soon for updates. The FDA granted fast track designation to SAR446597, an intravitreal gene therapy for the treatmentā¦
#ophthalmology
#eye
#eyenews
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FDA fast tracks intravitreal gene therapy for geographic atrophy
Editorās note: This is a developing news story. Please check back soon for updates. The FDA granted fast track designation to SAR446597, an intravitreal gene therapy for the treatment of geographic atrophy due to age-related macular degeneration, according to a press release from Sanofi. The one-time injection is designed to target and inhibit two components of the complement pathway: C1s in the classical pathway and factor Bb in the alternative pathway. āThis dual-targeting approach potentially offers clinical advantages by providing sustained complement suppression within the
http://dlvr.it/TLzC1M
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reposted by
Jason Shepherd
5 months ago
While Iām a big fan of new technologies that help us study human biologyā¦thereās still no replacement for animal research! See my article in Gen News.
www.genengnews.com/topics/trans...
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Why We Still Need Animal Research in a World of AI and Organoids
The portrayal of uncaring scientists without any thought for the animals being used in their research is far from the truth.
https://www.genengnews.com/topics/translational-medicine/why-we-still-need-animal-research-in-a-world-of-ai-and-organoids/
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Everyone, please comment to the FDA about the importance of animal research!!
www.fda.gov/news-events/...
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FDA-NIH Workshop: Reducing Animal Testing
The FDA is hosting a workshop on reducing animal testing. The workshop is open to current FDA and NIH employees.
https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/fda-nih-workshop-reducing-animal-testing-07072025
5 months ago
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Gordon research conference for eye movements! Not sure who to give credit to for the picture, Michele gets credit for the beards!
5 months ago
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reposted by
Dr. Jan Zimmermann
5 months ago
www.cell.com/cell/abstrac...
!!!
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Single-neuron projectomes of macaque prefrontal cortex reveal refined axon targeting and arborization
The reconstruction of whole-brain projectomes of 2,231 single neurons from the macaque prefrontal cortex reveals refined axon targeting and arborization, providing a structural foundation for primate-...
https://www.cell.com/cell/abstract/S0092-8674(25)00639-7
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reposted by
bioRxiv Neuroscience
5 months ago
Projection Targeting with Phototagging to Study the Structure and Function of Retinal Ganglion Cells
https://www.biorxiv.org/content/10.1101/2025.06.25.661576v1
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reposted by
Sonica Saraf
5 months ago
In many brain areas, neuronal tuning is heterogeneous. But how does this diversity help behavior? We show how tuning diversity shapes representational geometry and boosts coding efficiency for perception in our new preprint:
www.biorxiv.org/content/10.1...
(w/
@sueyeonchung.bsky.social
&Tony Movshon)
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Variations in neuronal selectivity create efficient representational geometries for perception
Our visual capabilities depend on neural response properties in visual areas of our brains. Neurons exhibit a wide variety of selective response properties, but the reasons for this diversity are unkn...
https://www.biorxiv.org/content/10.1101/2025.06.26.661754v1
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reposted by
5 months ago
Thoughts on this? >> Cell and Gene Therapy Market Size Rapidly Approaching $117.46 Billion by 2034 - BioSpace >> Comment below!
#strategy
#competitiveintelligence
#marketing
#healthcare
#pharmaceutical
#biotech
#pharma
#competitivemarketing
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Cell and Gene Therapy Market Size Rapidly Approaching $117.46 Billion by 2034 - BioSpace
Cell and Gene Therapy Market Size Rapidly Approaching $117.46 Billion by 2034Ā Ā BioSpace
http://dlvr.it/TLYQ3n
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reposted by
5 months ago
Thoughts on this? >> Adeno-Associated Virus Vector-Based Gene Therapy Market Report 2025-2034 - openPR.com >> Comment below!
#strategy
#competitiveintelligence
#marketing
#pharmaceutical
#competitivemarketing
#healthcare
#biotech
#pharma
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Adeno-Associated Virus Vector-Based Gene Therapy Market Report 2025-2034 - openPR.com
Adeno-Associated Virus Vector-Based Gene Therapy Market Report 2025-2034Ā Ā openPR.com
http://dlvr.it/TLYQ3x
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reposted by
Science X / Phys.org
6 months ago
New findings detail how structural changes in the VP1 protein of AAV vectors enable genome release, informing the design of safer and more efficient gene therapy delivery systems.
doi.org/g9qttr
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Unlocking the secrets of gene therapy delivery: New insights into genome ejection from AAV vectors
A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene therapy.
https://medicalxpress.com/news/2025-06-secrets-gene-therapy-delivery-insights.html?utm_source=bsky.app&utm_medium=social&utm_campaign=v2
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reposted by
HarishYaa
6 months ago
The Journey of Cell and Gene Therapy to Patients Read more :
bi-journal.com/journey-of-c...
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The Journey Of Cell And Gene Therapy To Patients
Cell and gene therapy is revolutionizing treatment, yet faces tough regulatory, clinical, and commercialization challenges in real-world adoption.
https://bi-journal.com/journey-of-cell-and-gene-therapy-to-patients/
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reposted by
Kavli Institute for Nanoscience Discovery, University of Oxford
6 months ago
#TuesdayReads
: Read the new review from the Wade-Martins Group exploring how gene therapy strategies are being developed to target key mechanisms in Parkinsonās disease. š
www.cell.com/molecular-th...
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Recent developments in gene therapy for Parkinsonās disease
Wade-Martins and colleagues outline current gene therapy approaches under development for the treatment of Parkinsonās disease (PD). The authors discuss the progress of several strategies targeting di...
https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(25)00204-7?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS1525001625002047%3Fshowall%3Dtrue
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