Science Translational Medicine

Don't miss the new issue of #ScienceTranslationalMedicine! A long-term AI risk model for breast cancer shows promise in primary prevention, next-generation imaging molecules could realize noninvasive tests for kidney fibrosis, and more. scim.ag/4eSv6GY

A new study employs AAV vectors and RNA-lipid nanoparticles to deliver a prime editor to correct citrullinemia type I in neonatal and juvenile mice, highlighting the technology’s potential for genetic liver disorders. @schwanklab @UZH_IPT #GeneTherapy scim.ag/49BakIl

Prime editing that targets a mutant allele in the gene SCN1A achieves efficient editing and reduces the frequency of febrile seizures in a mouse model of generalized epilepsy with febrile seizures plus (#GEFS+). @schwanklab @KisslingLucas @UZH_IPT scim.ag/4dsybLG

Leveraging adenine #BaseEditing, researchers demonstrate high editing efficiency of mutant SCN1A in a newborn mouse model of #DravetSyndrome and show it can lower seizure frequency while boosting survival. @davidrliu @broadinstitute scim.ag/4tBdXVM

A clinical trial of infants living with #HIV shows that broadly neutralizing antibodies are safe when combined with early antiretroviral therapy, underscoring the need for dedicated pediatric trials in the quest for ART-free remission. @IUDeptPeds scim.ag/4ezoPjg

Congrats to the Sunwoo Lab for their recent publication in @ScienceTM! 👏🔬 The @JohnSunwooMD Lab and collaborators, including Dr. Jennifer @FoltzPhd at Wash U, have identified a cytotoxic tissue-resident NK cell subset, characterized by the expression of CD39, CD49a, and CD103.

I'm very excited to see this work published in @ScienceTM . Proteogenomic studies have grown very rapidly in plasma, with almost every other context being completely ignored along the way. We find that even within closely related contexts (plasma and CSF), (1/3)

@DanWestern2 new @ScienceTM paper (science.org/doi/10.1126/sc…) demonstrates that CSF proteogenomics captures disease‑relevant pathways missed in plasma, uncovering 400+ protein–trait associations across AD, PD, MS, and psychiatric disorders. Less accessible ≠ less important.

Excited to share our paper in @ScienceTM! Together with @Francesca_Pietr as co-first author, we used in vivo prime editing to correct a pathogenic SCN1A mutation in a GEFS+ mouse model. Huge thanks to all co-authors for their work! doi.org/10.1126/scitra…

Our latest paper is out @ScienceTM! Natural killer cell immunotherapy reverses lung fibrosis by eliminating senescent fibroblasts | Science Translational Medicine science.org/doi/10.1126/sc… #Immunotherapy #Fibrosis #Aging #Senescence #NKcell

Researchers have discovered that #glioblastoma relies on the receptor uPAR for tumor initiation and growth and showed uPAR CAR T cells slow tumor growth in mice by neutralizing both cancer cells and supportive cells in the TME. @McMasterU @sheilasinghlab scim.ag/48Zg52u

A multiplexed platform named NeuroSense enables near real-time monitoring of CSF biomarkers in patients in the neurological ICU, potentially supporting timelier diagnosis and intervention. @Mahla_Poudineh @ShriyaSrinivas3 @jbernstock1 @WaterlooENG scim.ag/3Pg8xBK

A trio of papers highlights promising gene editing therapies, a near-real-time platform could support timelier monitoring of cerebrospinal fluid in neurocritical care, and more this week in #ScienceTranslationalMedicine. scim.ag/4uEt0iw

A new study delineates how the RNA-binding protein RBPMS correlates with #AML severity and drives the self-renewal of leukemic stem cells by stabilizing FOXO1, and presents a selective inhibitor that displays promising effects in mice. @CAS__Science scim.ag/4wwlpo6

New work ties traumatic heterotopic ossification (HO) to deposits of extracellular traps (ETs) from myeloid cells in traumatic musculoskeletal injury sites, and shows that blocking ETs can inhibit HO lesions in mice. @Ukehamburg scim.ag/42TSSel

A longitudinal study of #influenza vaccinees reveals that #FluMist—the only FDA-approved intranasal flu vaccine—promotes robust memory B cell responses in the upper respiratory tract that persist for 6 months. @profshanecrotty @Hannah_Stacey_ @ljiresearch scim.ag/49e3EQ6

A unique combination therapy that refashions #rapamycin into a polymeric nanoparticle delivery vehicle for ponatinib can release both drugs to shrink venous malformations in mice with a single dose. @BostonChildrens scim.ag/48Mlk5m

A clinical trial of infants living with #HIV shows that broadly neutralizing antibodies are safe when combined with early antiretroviral therapy, underscoring the need for dedicated pediatric trials in the quest for ART-free remission. @IUDeptPeds scim.ag/4ezoPjg

A new clinical trial reveals that combining antiretroviral therapy with anti-HIV antibodies is safe in infants, scientists refashion rapamycin into a dual therapy and drug delivery tool for venous malformations, and more in #ScienceTranslationalMedicine. scim.ag/4d8T9yT

Plasma and cerebrospinal fluid samples yield widely diverging protein quantitative trait loci in neurological disorders, shows new work that highlights the importance of trait-relevant contexts during protein and drug discovery. @ccrugom @washumedicine scim.ag/4t6XFny