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Showing 1–3 of 3 results

Advanced filters: Author: Congsheng Cheng Clear advanced filters

AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice

Amado et al. develop a gene therapy for sporadic ALS using motor neuron-targeting AAVs to deliver RNAi targeting ataxin-2. In a mouse model, survival, strength, and disease-related pathology are improved; and human motor neurons are strongly transduced.

Defne A. Amado
Ashley B. Robbins
Beverly L. Davidson
ResearchOpen Access25 Jun 2025
Nature Communications

Volume: 16, P: 1-17
Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching

Dawicki-McKenna and Felix et al comprehensively map binding and alternative splicing by PTBP2 in human brain and neurons, thus identifying splice switching therapeutic strategies for the neurodevelopmental disorder associated gene SYNGAP1.

Jennine M. Dawicki-McKenna
Alex J. Felix
Benjamin L. Prosser
ResearchOpen Access06 May 2023
Nature Communications

Volume: 14, P: 1-20
Iron limitation-induced endophytic Ammoniphilus assemblage promotes root apoplastic iron remobilization by attenuation of salicylic acid pathways

Lin Zhu
Aijing Zhang
Cheng Zhou
ResearchOpen Access01 Jul 2025
npj Biofilms and Microbiomes

Volume: 11, P: 1-17