Results 301 to 310 of about 12,497,887 (407)

Gene therapy for neurological disorders: progress and prospects

Nature reviews. Drug discovery, 2018
Benjamin E. Deverman, B. Ravina, K. Bankiewicz, S. Paul, D. Sah   +4 more
semanticscholar   +1 more source

Cellular transplantation and gene therapy. [PDF]

, 1993
Ildstad, ST, Ricordi, C, Starzl, TE
core  

Class IIa HDACs forced degradation allows resensitization of oxaliplatin‐resistant FBXW7‐mutated colorectal cancer

Molecular Oncology, EarlyView.
HDAC4 is degraded by the E3 ligase FBXW7. In colorectal cancer, FBXW7 mutations prevent HDAC4 degradation, leading to oxaliplatin resistance. Forced degradation of HDAC4 using a PROTAC compound restores drug sensitivity by resetting the super‐enhancer landscape, reprogramming the epigenetic state of FBXW7‐mutated cells to resemble oxaliplatin ...
Vanessa Tolotto, Nicolò Gualandi, Ylenia Cortolezzis, Raffaella Picco, Monica Colitti, Francesca D'Este, Mariachiara Gani, Wayne W. Hancock, Giovanni Terrosu, Cristina Degrassi, Francesca Agostini, Claudio Brancolini, Luigi E. Xodo, Eros Di Giorgio   +13 more
wiley   +1 more source

Astrocyte-neuron combined targeting for CYP46A1 gene therapy in Huntington's disease. [PDF]

Acta Neuropathol Commun
Parsai LH, Chali F, Subashi E, Zeitouny C, Rey E, Berniard A, Bitton W, Urli L, Rousselot L, Sarrazin N, Blouin V, Den Dunnen WFA, Michaelsen-Preusse K, Korte M, Alves S, Cartier N.   +15 more
europepmc   +1 more source

In vitro models of cancer‐associated fibroblast heterogeneity uncover subtype‐specific effects of CRISPR perturbations

Molecular Oncology, EarlyView.
Development of therapies targeting cancer‐associated fibroblasts (CAFs) necessitates preclinical model systems that faithfully represent CAF–tumor biology. We established an in vitro coculture system of patient‐derived pancreatic CAFs and tumor cell lines and demonstrated its recapitulation of primary CAF–tumor biology with single‐cell transcriptomics ...
Elysia Saputra, Shamsudheen Karuthedath Vellarikkal, Lixia Li, Hong Sun, Khoa Nguyen, Amber Montano, Suchitra Natarajan, Federica Piccioni, Alex Michael Tamburino, Xin Yu, Aleksandra Katarzyna Olow   +10 more
wiley   +1 more source

Advancing Gene Therapy for Phenylketonuria: From Precision Editing to Clinical Translation. [PDF]

Int J Mol Sci
Yu I, Jeong J.
europepmc   +1 more source

Gene therapy to promote thromboresistance: Local overexpression of tissue plasminogen activator to prevent arterial thrombosis in an in vivo rabbit model

, 1999
Jacob M. Waugh, Maan Kattash, J. Li, E. Yüksel, Michael Kuo, M. Lussier, Adam B. Weinfeld, Renu Saxena, E. D. Rabinovsky, Swan N. Thung, Savio L.C. Woo, Saleh M. Shenaq   +11 more
openalex   +1 more source

Identifying prognostic targets in metastatic prostate cancer beyond AR

FEBS Open Bio, EarlyView.
Genome‐wide functional screens combined with a large gene expression database and clinical outcomes can identify new therapeutic vulnerabilities in prostate cancer. Eight potentially druggable targets demonstrated strong dependency in cell lines, were associated with worse prognosis clinically, and showed evidence of protein expression in prostate ...
Emily Feng, Eric Feng, Tracy Berg, Isabella S. Nguyen, Lilac G. Nguyen, William Chen, Meng Zhang, David Quigley, Marina Sharifi, Haolong Li, Ilsa Coleman, Peter S. Nelson, Martin Sjöström, Shuang G. Zhao   +13 more
wiley   +1 more source

Significance of gene therapy in neurodegenerative diseases. [PDF]

Front Neurosci
Wang L, Ma L, Gao Z, Wang Y, Qiu J.
europepmc   +1 more source