Science Translational Medicine weekly highlights

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Science Translational Medicine: At the intersection of science, engineering and medicine.
Updated: 1 day 13 hours ago

RNA-LNP–mediated in vivo prime editing corrects disease phenotypes in a mouse model of citrullinemia type I | Science Translational Medicine

Wed, 13/05/2026 - 16:01
Prime editing via RNA-LNP corrects multiple pathogenic ASS1 mutations and restores ureagenesis and survival in a mouse model of citrullinemia type I.
Categories: Science

Sensory nerves protect against preclinical tendinopathic changes through FGF1 signaling | Science Translational Medicine

Wed, 13/05/2026 - 16:01
Somatosensory neurons prevent tendon degeneration by FGF1-mediated mesenchymal and macrophage regulation.
Categories: Science

A platform for near real-time and multiplexed monitoring of cerebrospinal fluid biomarkers and flow in neurocritical care | Science Translational Medicine

Wed, 13/05/2026 - 16:01
NeuroSense enables near real-time, multimodal monitoring of external ventricular drains in neurocritical care.
Categories: Science

uPAR is highly expressed in recurrent glioblastoma and represents a candidate CAR T cell target | Science Translational Medicine

Wed, 13/05/2026 - 16:01
The urokinase receptor uPAR is highly expressed in recurrent glioblastoma and is targetable using anti-uPAR chimeric antigen receptor T cells.
Categories: Science

Prime editing of a pathogenic Scn1a allele ameliorates seizure phenotypes in a GEFS+ mouse model | Science Translational Medicine

Wed, 13/05/2026 - 16:01
Prime editing corrects a pathogenic human SCN1A mutation in mice, reducing seizure frequencies and improving survival in this GEFS+ model.
Categories: Science

In vivo adenine base editing ameliorates Dravet syndrome phenotypes in a mouse model | Science Translational Medicine

Wed, 13/05/2026 - 16:01
Through direct correction of a mutation causing Dravet syndrome, adenine base editing alleviates epileptic and premature mortality phenotypes in mice.
Categories: Science

Natural killer cell immunotherapy reverses lung fibrosis by eliminating senescent fibroblasts | Science Translational Medicine

Wed, 13/05/2026 - 16:01
NKG2A checkpoint inhibition promotes lung fibrosis resolution.
Categories: Science

Erratum for the Research Article “Inhibiting the oncogenic translation program is an effective therapeutic strategy in multiple myeloma” | Science Translational Medicine

Wed, 29/04/2026 - 16:01
In the experiment depicted in Fig. 5C of the Research Article “Inhibiting the oncogenic translation program is an effective therapeutic strategy in multiple myeloma,” by S. Manier et al., MYC (57 kDa) and MAF (48 kDa) were probed on the same membrane, and in the originally published MAF column, both bands were showing. The authors have since corrected the figure to clarify that the MAF band is accurately presented at 48 kDa. No data were affected, and the conclusions have not changed.
Categories: Science

Erratum for the Research Article “Targeted disruption of the BCL9/β-catenin complex inhibits oncogenic Wnt signaling” | Science Translational Medicine

Wed, 29/04/2026 - 16:01
After publication of the Research Article “Targeted disruption of the BCL9/β-catenin complex inhibits oncogenic Wnt signaling” by K. Takada et al., the authors identified errors in fig. S7B, where two pairs of micrographs were inadvertently reused or misplaced. The sample 2 image for the SAH-BCL9MUT–treated group was a 180° rotation with partial duplication of sample 1 and has been replaced with the correct micrograph. For the vehicle-treated condition, the sample 1 and sample 2 images were inadvertently interchanged and have been corrected. Other data and the conclusions of the paper are not affected.
Categories: Science

Local B cell immunity and durable memory after live-attenuated influenza intranasal vaccination of humans | Science Translational Medicine

Wed, 29/04/2026 - 16:01
Longitudinal nasopharyngeal sampling reveals local influenza-specific B cell responses after intranasal, but not intramuscular, vaccination.
Categories: Science

Low overlap of plasma and CSF protein quantitative trait loci affects protein discovery for neurological disease | Science Translational Medicine

Wed, 29/04/2026 - 16:01
Genomic regulation of protein abundance differs substantially between human biofluids, affecting neurological disease–relevant target discovery.
Categories: Science

Clinical cure of chronic hepatitis B is associated with priming and perpetuation of hepatic CD4+ T cell responses | Science Translational Medicine

Wed, 29/04/2026 - 16:01
Samples from patients with chronic hepatitis B and a mouse model revealed a central role for CD4+ T cells in hepatitis B virus clearance.
Categories: Science

Adeno-associated virus gene therapy–mediated CCR5 blockade suppresses virus replication long term in SHIV-infected macaques | Science Translational Medicine

Wed, 29/04/2026 - 16:01
A single dose of an AAV vector encoding the CCR5-blocking antibody leronlimab drives suppression of viremia in SHIV-infected macaques.
Categories: Science

The internal limiting basement membrane inhibits functional engraftment of transplanted human retinal ganglion cells in vivo | Science Translational Medicine

Wed, 29/04/2026 - 16:01
Disrupting the ILM in vivo permits retinal integration of transplanted RGCs, enabling connectivity to light-sensing retinal circuits.
Categories: Science

Dipeptidyl aminopeptidase–like protein 6 regulates the INa-Ito balance influencing cardiac electrophysiology and arrhythmogenesis | Science Translational Medicine

Wed, 29/04/2026 - 16:01
DPP6 regulates the cardiac INa-Ito balance, which is aberrantly influenced by clinically relevant DPP6 gene variants.
Categories: Science

Erratum for the Focus “American medical education at a crossroads” | Science Translational Medicine

Wed, 22/04/2026 - 16:01
The original publication of the Focus “American medical education at a crossroads” by A. M. Feldman et al. omitted the disclosure that Marschall S. Runge was serving on the board of directors of Eli Lilly. The Acknowledgments section has been updated.
Categories: Science

Programming peripheral artery disease in diabetes | Science Translational Medicine

Wed, 22/04/2026 - 16:01
Reprogrammed TREM2+ macrophages drive nonresolving inflammation in peripheral artery disease associated with type 2 diabetes (Malhi et al., this issue).
Categories: Science

Peripheral blood transcriptional profiling predicts tumor subtype and neoadjuvant chemoimmunotherapy outcomes in human breast cancer | Science Translational Medicine

Wed, 22/04/2026 - 16:01
Transcriptomics of peripheral blood from patients with breast cancer during therapy identifies changes in immunity linked to therapeutic response.
Categories: Science

Expansion of stemlike HIV-specific CD8+ T cells and limited viral epitope diversity characterize durable posttreatment control of HIV | Science Translational Medicine

Wed, 22/04/2026 - 16:01
Stemlike HIV-specific CD8+ T cells and limited viral epitope diversity are associated with durable HIV remission after treatment interruption.
Categories: Science

Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of HNRNPH2-related neurodevelopmental disorder | Science Translational Medicine

Wed, 22/04/2026 - 16:01
ASO-mediated Hnrnph2 knockdown up-regulates Hnrnph1 and rescues multiple phenotypes in a mouse model of HNRNPH2-related neurodevelopmental disorder.
Categories: Science

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