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haoyin.bsky.social
Induced EndMT status enhances AAV6 or AVV9 transduction efficiency in Cardiac Endothelial Cell by 5~10 fold in vitro & ~3 fold in vivo

Vatalanib
siVEGFR1
siVEGFR2
siPECAM1
TGFβ2

Experimental myocardial infarction

#MolTherapy 2024
www.cell.com/molecular-th...
January 24, 2026 at 8:06 PM
labwaggoner.bsky.social
Review @MolTherapy Cancer Viroimmunotherapy Platforms Based on Varicella-Zoster Virus and Cytomegalovirus @mayoclinic.org
www.cell.com/molecular-th...
October 7, 2025 at 5:28 PM
haoyin.bsky.social
#LipidNanoParticle functionalized with Myomerger extracellular domain for #SkeletalMuscle targeting (+ liver detargeting by 85% at 24 h i.m 😎) delivery of nucleic acids

⏬ innate immune response in lymph node & muscle by 24 h i.m.

#MyoSky

#MolTherapy 2026
www.cell.com/molecular-th...
February 19, 2026 at 8:02 PM
labwaggoner.bsky.social
Review: Emerging Novel Combined CAR-NK Cell Therapies in Cancer Treatment: Finding a Dancing Partner
@MolTherapy
doi.org/10.1016/j.ym...
January 4, 2025 at 7:20 PM
mattmuscleguy.bsky.social
Happy to be a part of another great collaboration with @JimDowling for this new @MolTherapy #MTNA paper: microRNA-133a as an indicator of disease progression and treatment response in X-linked myotubular myopathy. #Myoblue. 1/n tinyurl.com/yc5bdbfe
microRNA-133a as an indicator of disease progression and treatment response in X-linked myotubular myopathy
Dowling and colleagues identify miR-133a as a non-invasive biomarker for monitoring disease progression and treatment response in X-linked myotubular myopathy (XLMTM). Their study reveals its correlat...
tinyurl.com
March 10, 2025 at 12:20 AM
thatsregrettab1.bsky.social
Journal @MolTherapy took their time on this, but after 4 years made the right decision to retract. Probably a paper mill product.

Once again, despite the 🚨WARNINGS🚨 of SciGuardians and @weldeiry.bsky.social, the Editor in Chief credited the PubPeer post.

pubpeer.com/publications...
June 20, 2025 at 12:47 AM
mattmuscleguy.bsky.social
New #MolTherapy paper: Development of an AAV-delivered microRNA gene therapy for Myotonic Dystrophy Type 1. #Myoblue tinyurl.com/mrjrue3f
Development of an AAV-delivered microRNA gene therapy for Myotonic Dystrophy Type 1
An AAV-mediated gene therapy for myotonic dystrophy type 1 using a novel muscle-tropic capsid and promoter to express a DMPK-targeting miRNA effectively reduces DMPK expression and nuclear RNA foci, a...
tinyurl.com
September 4, 2025 at 4:25 PM
haoyin.bsky.social
p21-ATD🐭for p21+ senescent cell tracking & elimination in #Aging & Liver injury
#HereditaryTyrosinemia Fah1 KO🐭

Senescent hepatocytes are mainly p21+ p16-

p21 cell ablation
⏬tyrosinemic liver-derived CCL2 & ⏫donor hepatocyte engraftment

#MolTherapy 2024
www.sciencedirect.com/science/arti...
October 28, 2025 at 11:37 AM
mattmuscleguy.bsky.social
New #MolTherapy paper: AAV-microutrophin gene therapy confers long-term cardioprotection against pharmacologic and exercise-induced injury in dystrophin deficiency. #Myoblue tinyurl.com/ypdkckmc
AAV-microutrophin gene therapy confers long-term cardioprotection against pharmacologic and exercise-induced injury in dystrophin deficiency
AAV-microutrophin gene therapy confers durable cardioprotection during heightened cardiac workload in the Duchenne muscular dystrophy mouse. In novel drug- and exercise-induced cardiac stress assays, ...
tinyurl.com
July 17, 2025 at 1:29 PM
mattmuscleguy.bsky.social
New #MolTherapy paper: Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy. #DMD #Myoblue tinyurl.com/4f5tj2xn
Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy
High-dose microdystrophin gene therapy improves muscle pathology in DMDmdx rats but can induce dose-dependent cardiac toxicity, including arrhythmias and sudden death, in both DMDmdx and wild-type rat...
tinyurl.com
October 23, 2025 at 8:33 PM
mattmuscleguy.bsky.social
Congrats to #ronald_cohn & colleagues for their new #MolTherapy paper: Template-assisted sequence knock-in rescues skeletal and cardiac muscle function in a deletion model of Duchenne muscular dystrophy. tinyurl.com/5x2sk6j2
Template-assisted sequence knock-in rescues skeletal and cardiac muscle function in a deletion model of Duchenne muscular dystrophy
Cohn and colleagues demonstrate TASK, a CRISPR-mediated DNA integration strategy for full-length dystrophin restoration in a deletion mouse model of DMD. Precise integration of the missing exons into ...
tinyurl.com
May 8, 2025 at 6:30 PM
haoyin.bsky.social
Systematic screening of 2042👤miRNAs➡️
7 new miRs (miR-323a-3p, -449b-5p, -491-3p, -892b, -1827, -4774-3p, -5681b) that⏬#SmoothMuscleCell (of multi-vascular beds) proliferation w/o cytotoxicity

miR-323a-3p & -449b-5p validated on ex vivo👤vein graft

#MolTherapy 2024
www.cell.com/molecular-th...
January 12, 2025 at 12:46 PM
freddyeescorcia.bsky.social
🚨New pub alert!🚨

Excited to share our @MolTherapy pub resulting from a massive collaboration w friends and colleagues Rosa Nguyen & @RPatelMDPHD & their teams!

We explore GPC2-targeted #radiopharmaceuticals #theranostics CAR T cell therapy in models of #neuroblastoma!

👇🧵
1/9
June 17, 2026 at 10:43 AM
mattmuscleguy.bsky.social
New #MolTherapy paper: AUF1 therapy blocks atrophy, promotes regeneration, re-innervation and strength for severe muscle injury. #Myoblue tinyurl.com/3bmnxr8d
AUF1 therapy blocks atrophy, promotes regeneration, re-innervation and strength for severe muscle injury
Abbadi, Schneider and colleagues demonstrate that systemic or local delivery of RNA binding protein AUF1 to muscle, before or after severe muscle injury, strongly reduces atrophy, accelerates muscle r...
tinyurl.com
February 26, 2026 at 5:36 PM
mattmuscleguy.bsky.social
New #MolTherapy paper: Delpacibart Etedesiran Improves the Molecular Pathology of Myotonic Dystrophy Type 1 in the Phase 1/2 MARINA® Study. #Myoblue tinyurl.com/3kbu3ty3
Delpacibart Etedesiran Improves the Molecular Pathology of Myotonic Dystrophy Type 1 in the Phase 1/2 MARINA® Study
Del-desiran, an antibody-oligonucleotide conjugate targeting DMPK mRNA, reduced toxic DMPK transcript levels, restored functional MBNL, and corrected mis-splicing in patients with myotonic dystrophy 1...
tinyurl.com
March 12, 2026 at 5:55 PM
parkerici.bsky.social
CAR T-cell therapy isn’t just a breakthrough—it’s a lifeline. A new PICI-supported review in @MolTherapy by Carl June, MD, Director of the PICI Center at @PennMedicine, and team traces the decades of research that made engineered immune cells a powerful cancer treatment. cell.com/molecular-th...
From Concept to Cure: The Evolution of CAR-T Cell Therapy
This review provides a comprehensive overview of the historical evolution of CAR-T cell therapy, highlighting key advancements that have shaped its success. It details currently approved CAR-T cell tr...
cell.com
March 17, 2025 at 8:50 PM
sahakris.bsky.social
Looking to control #CRISPR genome editing for therapeutic purposes? Check out our new @MolTherapy Review written by @omgitsnamita and me. Thank you to @somaticediting @CMaT_ERC, in particular @Roy_Lab_Thinks, for very helpful discussions.
November 14, 2024 at 4:44 AM
mattmuscleguy.bsky.social
Congrats to #EricTWang & colleagues on their new #MolTherapy paper: Enhanced antisense oligonucleotide delivery reveals that transcript turnover impacts apparent splicing rescue in myotonic dystrophy. #Myoblue tinyurl.com/mt75rmaw
Enhanced antisense oligonucleotide delivery reveals that transcript turnover impacts apparent splicing rescue in myotonic dystrophy
Wang and colleagues show that a cyclic peptide-conjugated antisense oligonucleotide rapidly rescues mis-splicing in a mouse model of myotonic dystrophy. Computational modeling and in vivo nascent RNA ...
tinyurl.com
May 21, 2026 at 4:16 PM
labwaggoner.bsky.social
Review @MolTherapy on Four Decades of Adenovirus Gene Transfer Vectors: History and Current Use
www.cell.com/molecular-th...
April 8, 2025 at 12:52 PM