Genetic Therapy and Molecular Targeted Therapy in Oncology: Safety, Pharmacovigilance, and Perspectives for Research and Clinical Practice. [PDF]
Orzetti S+8 more
europepmc +1 more source
Gene Therapy for Human Genetic Disease? [PDF]
Elizabeth F. Neufeld, Charles C. Sweeley
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There is an unmet need in metastatic breast cancer patients to monitor therapy response in real time. In this study, we show how a noninvasive and affordable strategy based on sequencing of plasma samples with longitudinal tracking of tumour fraction paired with a statistical model provides valuable information on treatment response in advance of the ...
Emma J. Beddowes+20 more
wiley +1 more source
Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models. [PDF]
Chilcott EM+3 more
europepmc +1 more source
Comments on important genetic topics from papers in other journals: Prospects for homologous recombination in human gene therapy [PDF]
J C K Barber
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Loss of the frequently mutated chromatin remodeler ARID1A, a subunit of the SWI/SNF cBAF complex, results in less open chromatin, alternative splicing, and the failure to stop cells from progressing through the cell cycle after DNA damage in bladder (cancer) cells. Created in BioRender. Epigenetic regulators, such as the SWI/SNF complex, with important
Rebecca M. Schlösser+11 more
wiley +1 more source
Adenine base editing of the DUX4 polyadenylation signal for targeted genetic therapy in facioscapulohumeral muscular dystrophy. [PDF]
Šikrová D+5 more
europepmc +1 more source
Genetically targeted research & therapeutics: Antisense & gene therapy [PDF]
openalex +1 more source
Chronic TGF‐β exposure drives epithelial HCC cells from a senescent state to a TGF‐β resistant mesenchymal phenotype. This transition is characterized by the loss of Smad3‐mediated signaling, escape from senescence, enhanced invasiveness and metastatic potential, and upregulation of key resistance modulators such as MARK1 and GRM8, ultimately promoting
Minenur Kalyoncu+11 more
wiley +1 more source
With newer tools for gene editing, is it time to revisit genetic therapy for cystic fibrosis? [PDF]
Gupta A, Gupta A, Boyd L.
europepmc +1 more source