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...

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

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...

New #MolTherapy paper: AUF1 therapy blocks atrophy, promotes regeneration, re-innervation and strength for severe muscle injury. #Myoblue tinyurl.com/3bmnxr8d

#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...

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...

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...

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...

New #MolTherapy paper: Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy. #DMD #Myoblue tinyurl.com/4f5tj2xn

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...

New #MolTherapy paper: Development of an AAV-delivered microRNA gene therapy for Myotonic Dystrophy Type 1. #Myoblue tinyurl.com/mrjrue3f

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, ...

New #MolTherapy paper: AAV-microutrophin gene therapy confers long-term cardioprotection against pharmacologic and exercise-induced injury in dystrophin deficiency. #Myoblue tinyurl.com/ypdkckmc

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 ...

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

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...