Boosting a key brain protein could help treat Rett syndrome Researchers have discovered a new way to increase a key brain protein damaged in Rett syndrome, a rare genetic disorder that affects thousands of children worldwide. Early studies in mice and patient-...

A new gene-targeting strategy that boosts a critical brain protein may offer a future path toward treating #RettSyndrome. From H Zoghbi lab, @harinipt6 et al @bcmhouston.bsky.social @bcmgenetics.bsky.social #ScienceTM @rettsyndrome.bsky.social www.sciencedaily.com/releases/202...

AAV8 gene therapy and dietary insults together precipitate cholestatic liver disease in a mouse model of X-linked myotubular myopathy Purified diet feeding and AAV8 therapy are precipitants of cholestatic liver injury in a mouse model of X-linked myotubular myopathy.

Congrats to Jim Dowling and colleagues for their new #ScienceTM paper: AAV8 gene therapy and dietary insults together precipitate cholestatic liver disease in a mouse model of X-linked myotubular myopathy. #Myoblue tinyurl.com/ctdha2wc

Senolytic CAR T cell
NKG2DL-targeting

Irradiated or Aged🐭
⏫Adipocyte size
⏫Bone volume
⏫Myofiber size
⏫Physical activity

Rhesus/Cynomolgus macaques 19~22 y/o
A single dose of 1 M hNKG2D-CAR T cells
2-mo later⏬Senescence in Fat, Muscle, Liver, Kidney

#ScienceTM 2023
www.science.org/doi/10.1126/...

Water-handling or H2O2-transporting capacity of Aquaporin 1 in Cardiomyocyte is important to Cardiac Hypertrophy

AQP1 KO🐭+TAC

In isolated cardiomyocytes, AQP1 KO or AQP1i ⏬hypertrophy induced by AngII/PE/ET1/ISO, Not that by hypo-osmotic stress

#ScienceTM 2020
www.science.org/doi/10.1126/...

#DiabeticRetinopathy

EC LRG1 ⏫Pericyte TGFβ▶️
Hypercontractile & matrix phenotype

Lrg1 KO in hyperglycemic🐭 rescues
👉Retinal vascular permeability
👉⬇️Density φ Oxygenation in deep vasc plexus
👉⬇️ERG b-wave amp

@giuliaderossi.bsky.social #ScienceTM 2025
www.science.org/doi/10.1126/...

Breast cancer clues hint at response to therapy in animal models The findings bring us one step closer to improving ER+ breast cancer patients’ response to therapy.

Some #breastCancers provide clues indicating whether they are susceptible to #therapy, helping inform treatment decisions. E Chang et al. #ScienceTM @bcmhouston.bsky.social #BCMCancerCenter blogs.bcm.edu/2025/09/18/f...

#SpatialTranscriptomics 🐭 #Stroke

Stroke▶️
⏫Microglia/Macrophage Galectin-1/3/9➡️ Astrocyte/Oligodendrocyte CD44 at peri-infarct zone▶️
⏫remyelination
⏫neurobehavioral performance

Post-stroke benefits of LGALS9-expressing #ExtracellularVesicle

#ScienceTM 2024
www.science.org/doi/10.1126/...

Tumor-targeted cytokine delivery by engineered myeloid cells unlocks CAR T cell efficacy and endogenous T cell responses in glioblastoma @moltherapy.bsky.social
on
www.science.org/doi/10.1126/... #ScienceTM

Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy Antisense oligonucleotides delivered in utero can treat mouse models of SMA and can feasibly be administered to fetal lambs at midgestation.

Interesting new #ScienceTM work from #CharlotteSumner and colleagues: Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy. #Myoblue tinyurl.com/y47jxj3z

Pentatricopeptide repeat protein targeting CUG repeat RNA ameliorates RNA toxicity in a myotonic dystrophy type 1 mouse model AAV9-mediated systemic delivery of a protein that targets CUG repeat RNA ameliorates myotonia in a mouse model of myotonic dystrophy.

New #ScienceTM paper: Pentatricopeptide repeat protein targeting CUG repeat RNA ameliorates RNA toxicity in a myotonic dystrophy type 1 mouse model. #Myoblue tinyurl.com/4sf3rps8

Autologous #CellTherapy for 👤#ChronicThreateningLimbIschemia

CD16+ pro-arteriogenic monocyte
HB-EGF paracrine, likely +other factors
N=5
100~150 ml apheresis
30M cells im
25% cell retention by D3
⏫arteriole/capillary/peri-arteriolar macrophage by D10

#ScienceTM 2024
www.science.org/doi/10.1126/...

#EndothelialCell TGFβR2 is essential for autocrine VEGF-A (major source in injured🫁) & LRG1 to ⏫#MicrovascularRegeneration in🐭#ViralPneumonia #Influenza

Really a thought-provoking paper👏

I will re-think my ischemic limb data!

@vaughandy.bsky.social #ScienceTM 2024
www.science.org/doi/10.1126/...

Molecular Anatomy of #Aortitis

scRNAseq
22 #Aortitis vs 20 noninflamed #AscendingAorticAneurysm

Discovery Stem-like TCFhiIL7R+CD4+ T cell residing in aortic adventitial #TertiaryLymphoidStructure
Serially transplantable😈

#ScienceTM 2023
www.science.org/doi/full/10....

Oops, just noticed I missed a couple of slides out - make that 238 Refs #ScienceTM

Why does changing the font of my manuscripts from Arial to Times New Roman suddenly make them 'appear' more #ScienceTM?