CRISPR-Cas9: a preclinical and clinical perspective for the treatment of human diseases
G Sharma, AR Sharma, M Bhattacharya, SS Lee… - Molecular Therapy, 2021 - cell.com
At present, the idea of genome modification has revolutionized the modern therapeutic
research era. Genome modification studies have traveled a long way from gene …
research era. Genome modification studies have traveled a long way from gene …
Targeted genome editing tools for disease modeling and gene therapy
M Cai, Y Yang - Current gene therapy, 2014 - ingentaconnect.com
The development of custom-designed nucleases (CDNs), including zinc finger nucleases
(ZFNs) and transcription activator-like effector nucleases (TALENs), has made it possible to …
(ZFNs) and transcription activator-like effector nucleases (TALENs), has made it possible to …
[HTML][HTML] Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine
M Khoshandam, H Soltaninejad, M Mousazadeh… - Genes & …, 2024 - Elsevier
CRISPR/Cas9 is an effective gene editing tool with broad applications for the prevention or
treatment of numerous diseases. It depends on CRISPR (clustered regularly interspaced …
treatment of numerous diseases. It depends on CRISPR (clustered regularly interspaced …
NTLA-2001: opening a new era for gene therapy
T Cui, B Li, W Li - 2022 - academic.oup.com
Recently, Intellia Therapeutics, a gene-editing company co-founded by Nobel laureate
Jennifer Doudna, along with Regeneron Pharmaceuticals, created history and reported the …
Jennifer Doudna, along with Regeneron Pharmaceuticals, created history and reported the …
Genome-editing technologies for gene and cell therapy
ML Maeder, CA Gersbach - Molecular therapy, 2016 - cell.com
Gene therapy has historically been defined as the addition of new genes to human cells.
However, the recent advent of genome-editing technologies has enabled a new paradigm in …
However, the recent advent of genome-editing technologies has enabled a new paradigm in …
Genome editing in stem cells for disease therapeutics
M Song, S Ramakrishna - Molecular biotechnology, 2018 - Springer
Programmable nucleases including zinc finger nucleases, transcription activator-like effector
nucleases, and clustered regularly interspaced short palindrome repeats (CRISPR)/CRISPR …
nucleases, and clustered regularly interspaced short palindrome repeats (CRISPR)/CRISPR …
Targeted genome editing in acute lymphoblastic leukemia: a review
A Montaño, M Forero-Castro, JM Hernández-Rivas… - BMC …, 2018 - Springer
Background Genome editing technologies offers new opportunities for tackling diseases
such as acute lymphoblastic leukemia (ALL) that have been beyond the reach of previous …
such as acute lymphoblastic leukemia (ALL) that have been beyond the reach of previous …
Applications of CRISPR-Cas9 for advancing precision medicine in oncology: from target discovery to disease modeling
M Ravichandran, D Maddalo - Frontiers in Genetics, 2023 - frontiersin.org
The clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-
associated protein 9 (Cas9)(CRISPR/Cas9) system is a powerful tool that enables precise …
associated protein 9 (Cas9)(CRISPR/Cas9) system is a powerful tool that enables precise …
CRISPR-derived genome editing therapies: Progress from bench to bedside
The development of CRISPR-derived genome editing technologies has enabled the precise
manipulation of DNA sequences within the human genome. In this review, we discuss the …
manipulation of DNA sequences within the human genome. In this review, we discuss the …