[HTML][HTML] Programmable genome editing tools and their regulation for efficient genome engineering

TK Guha, A Wai, G Hausner - Computational and structural biotechnology …, 2017 - Elsevier
Targeted genome editing has become a powerful genetic tool for studying gene function or
for modifying genomes by correcting defective genes or introducing genes. A variety of …

CRISPR therapeutic tools for complex genetic disorders and cancer

S Baliou, M Adamaki… - International …, 2018 - spandidos-publications.com
One of the fundamental discoveries in the field of biology is the ability to modulate the
genome and to monitor the functional outputs derived from genomic alterations. In order to …

Therapeutic approaches in pancreatic cancer: recent updates

L Kumar, S Kumar, K Sandeep, SKS Patel - Biomedicines, 2023 - mdpi.com
Cancer is a significant challenge for effective treatment due to its complex mechanism,
different progressing stages, and lack of adequate procedures for screening and …

[HTML][HTML] In vivo genome editing as a potential treatment strategy for inherited retinal dystrophies

M Yanik, B Müller, F Song, J Gall, F Wagner… - Progress in retinal and …, 2017 - Elsevier
In vivo genome editing represents an emerging field in the treatment of monogenic
disorders, as it may constitute a solution to the current hurdles in classic gene addition …

CAR T cells and checkpoint inhibition for the treatment of glioblastoma

SH Shen, K Woroniecka, AB Barbour… - Expert Opinion on …, 2020 - Taylor & Francis
Introduction: Glioblastoma (GBM) is a highly aggressive brain tumor and is one of the most
lethal human cancers. Chimeric antigen receptor (CAR) T cell therapy has markedly …

Epigenetic targeting of granulin in hepatoma cells by synthetic CRISPR dCas9 epi-suppressors

H Wang, R Guo, Z Du, L Bai, L Li, J Cui, W Li… - … Therapy-Nucleic Acids, 2018 - cell.com
The CRISPR-associated Cas9 system can modulate disease-causing alleles both in vivo
and ex vivo, raising the possibility of therapeutic genome editing. In addition to gene …

Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients

B Diez, P Genovese, FJ Roman‐Rodriguez… - EMBO molecular …, 2017 - embopress.org
Gene targeting constitutes a new step in the development of gene therapy for inherited
diseases. Although previous studies have shown the feasibility of editing fibroblasts from …

Genome-wide CRISPR screening identifies DCK and CCNL1 as genes that contribute to gemcitabine resistance in pancreatic cancer

H Yang, B Liu, D Liu, Z Yang, S Zhang, P Xu, Y Xing… - Cancers, 2022 - mdpi.com
Simple Summary Pancreatic cancer is one of the most lethal cancers. Although complete
surgical resection is the only curative treatment for pancreatic cancer, a late diagnosis is …

Редактирование генома человека

ДВ Ребриков - Вестник Российского государственного …, 2016 - cyberleninka.ru
Быстро развивающиеся технологии редактирования генома из научно-
исследовательских лабораторий уверенно переходят в клиническую практику …

[HTML][HTML] Gene therapy and genome editing for primary immunodeficiency diseases

ZY Zhang, AJ Thrasher, F Zhang - Genes & Diseases, 2020 - Elsevier
In past two decades the gene therapy using genetic modified autologous hematopoietic
stem cells (HSCs) transduced with the viral vector has become a promising alternative …