Gene editing of human hematopoietic stem and progenitor cells: promise and potential hurdles

KR Yu, H Natanson, CE Dunbar - Human Gene Therapy, 2016 - liebertpub.com
Hematopoietic stem and progenitor cells (HSPCs) have great therapeutic potential because
of their ability to both self-renew and differentiate. It has been proposed that, given their …

[HTML][HTML] Advances in CRISPR/Cas-based gene therapy in human genetic diseases

SS Wu, QC Li, CQ Yin, W Xue, CQ Song - Theranostics, 2020 - ncbi.nlm.nih.gov
CRISPR/Cas genome editing is a simple, cost effective, and highly specific technique for
introducing genetic variations. In mammalian cells, CRISPR/Cas can facilitate non …

Cornerstones of CRISPR–Cas in drug discovery and therapy

C Fellmann, BG Gowen, PC Lin, JA Doudna… - Nature reviews Drug …, 2017 - nature.com
The recent development of CRISPR–Cas systems as easily accessible and programmable
tools for genome editing and regulation is spurring a revolution in biology. Paired with the …

Editing human hematopoietic stem cells: advances and challenges

SV Bhoopalan, JS Yen, RM Levine, A Sharma - Cytotherapy, 2023 - Elsevier
Genome editing of hematopoietic stem and progenitor cells is being developed for the
treatment of several inherited disorders of the hematopoietic system. The adaptation of …

The transformative potential of AI-driven CRISPR-Cas9 genome editing to enhance CAR T-cell therapy

A Boretti - Computers in Biology and Medicine, 2024 - Elsevier
This narrative review examines the promising potential of integrating artificial intelligence
(AI) with CRISPR-Cas9 genome editing to advance CAR T-cell therapy. AI algorithms offer …

[HTML][HTML] Strategies to overcome the main challenges of the use of CRISPR/Cas9 as a replacement for cancer therapy

MF Rasul, BM Hussen, A Salihi, BS Ismael, PJ Jalal… - Molecular Cancer, 2022 - Springer
CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats-associated protein
9) shows the opportunity to treat a diverse array of untreated various genetic and …

Diagnosis and therapy with CRISPR advanced CRISPR based tools for point of care diagnostics and early therapies

V Uppada, M Gokara, GK Rasineni - Gene, 2018 - Elsevier
Molecular diagnostics is of critical importance to public health worldwide. It facilitates not
only detection and characterization of diseases, but also monitors drug responses, assists in …

[HTML][HTML] PCR-Based Strategy for Introducing CRISPR/Cas9 Machinery into Hematopoietic Cell Lines

E González-Romero, C Martínez-Valiente… - Cancers, 2023 - mdpi.com
Simple Summary We used PCR-generated small CRISPR constructs to edit two genes
(IDH2 and MYBL2) in hard-to-transfect hemopoietic cells, which are central to the …

Refining strategies to translate genome editing to the clinic

TI Cornu, C Mussolino, T Cathomen - Nature medicine, 2017 - nature.com
Recent progress in developing programmable nucleases, such as zinc-finger nucleases,
transcription activator–like effector nucleases (TALENs) and clustered regularly interspaced …

[HTML][HTML] Imagine CRISPR cures

FD Urnov - Molecular Therapy, 2021 - cell.com
A decade of progress in sequencing and in CRISPR/Cas technologies has created a
situation without precedent in the history of medicine. Starting with an individual patient, next …