Results 231 to 240 of about 214,719 (303)

Clinical‐Grade Human Induced Pluripotent Stem Cell‐Derived Neural Precursor Cells Restore Motor Function and Preserve Striatal Integrity in a Quinolinic Acid‐Lesioned Rat Model of Huntington's Disease

Cell Proliferation, EarlyView.
Clinical‐grade HLA‐homozygous iPSC‐derived neural precursor cells restore motor function, rebuild striatal circuitry and reduce neuroinflammation in QA‐lesioned rats. These findings demonstrate robust neuronal replacement and microenvironment modulation, supporting their potential as a regenerative therapy for Huntington's disease.
Hyeonjoong Jeon, Il‐Shin Lee, Sanghun Lee, Hyo Chang Park, Beomsoo Kim, Hyun Sook Kim, Jihwan Song   +6 more
wiley   +1 more source

Suppressing microtubule detyrosination augments adeno-associated virus 2 endosomal escape and gene delivery. [PDF]

J Cell Sci
Tripathi S, Huda S, Kar J, Chandra D, Jayandharan GR, Mohan N.   +5 more
europepmc   +1 more source

Adeno-Associated Virus (AAV) [PDF]

, 2008
openaire   +1 more source

Hepatocyte TrkB Acts as a Gatekeeper Against MASH‐Related Liver Fibrosis by Suppressing the TGFβ/CCL2 Axis and Macrophage Infiltration

Cell Proliferation, EarlyView.
Hepatocyte TrkB is identified as a critical gatekeeper against MASH‐related fibrosis. Mechanistically, TrkB inhibits the TGFβ/SMAD3/FOS axis to suppress CCL2 secretion, thereby blocking pathogenic macrophage recruitment and ameliorating liver fibrosis.
Yueying Chen, Jiayi Wei, Shuxuan Li, Kefan Yin, Heming Wang, Yicheng Zhao, Shuqiang Weng, Xizhong Shen, Guangqi Song, Changfeng Zhu, Qunyan Yao, Ling Dong   +11 more
wiley   +1 more source

Heterogeneity in an adeno-associated virus transfection-based production process limits the production efficiency. [PDF]

Sci Rep
Ladd B, Tunmats S, Gräslund T, Daramola O, Rockberg J, Chotteau V.   +5 more
europepmc   +1 more source

A Specific tRNA Half, 3'tiRNA‐GlyGCC, Regulates Hypoxic Pulmonary Artery Smooth Muscle Cell Proliferation via Myrf‐Mediated Endoplasmic Reticulum Stress

Cell Proliferation, EarlyView.
A schematic diagram illustrating how 3′tiRNA‐GlyGCC promotes the endoplasmic reticulum stress (ERS) and proliferation in hypoxic pulmonary artery smooth muscle cells (PASMCs) by inhibiting the expression of myelin regulatory factor (Myrf), ultimately leading to pulmonary hypertension (PH).
Lixin Zhang, Xiaoyu Guan, Xiangrui Zhu, Yingying Hao, Ya Xu, Hao Yuan, Danni Gao, Hongxia Du, Jiayu Li, Weiwei Cao, Cui Ma, Xiaoying Wang   +11 more
wiley   +1 more source

The Role of SAMHD1 in Viral Resistance and Transduction Efficiency Challenges in Pediatric Hematological Malignancies: Mechanistic Insights and Clinical Perspectives

European Journal of Haematology, EarlyView.
SAMHD1 regulates intracellular dNTP pools, influencing lentiviral transduction, gene therapy efficiency, and disease progression in pediatric hematological malignancies. Integrated bioinformatics and targeted strategies, including CRISPR and pharmacological inhibition, highlight its therapeutic potential.
Waseem Alzamzami
wiley   +1 more source

Correction to Accelerated Adeno Associated Virus Upstream Process Development From High-Throughput Systems to Clinical Scale. [PDF]

Biotechnol Bioeng
europepmc   +1 more source

Identification and Phylogenetic Characterisation of Novel Adeno-Associated Virus Capsids in Non-Human Primate Tissues. [PDF]

Cell Prolif
Zhu L, Xu K, Ding Y, Liu K, Liu J, Hou Z, Niu R, Yang N, Qin H, Hu B, Zhang Y, Li W.   +11 more
europepmc   +1 more source

Bringing Gene Therapy Into Real World Clinical Practice

Haemophilia, EarlyView.
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach, Pratima Chowdary, Margareth C. Ozelo   +2 more
wiley   +1 more source