Recent advances and applications of the CRISPR-Cas system in the gene therapy of blood disorders
Clinical gene therapy has faced numerous challenges and obstacles in the last few years.
However, it has made significant advances in treatment and is now in clinical trials and on …
However, it has made significant advances in treatment and is now in clinical trials and on …
Multiplex CRISPR/Cas9-based genome editing in human hematopoietic stem cells models clonal hematopoiesis and myeloid neoplasia
Hematologic malignancies are driven by combinations of genetic lesions that have been
difficult to model in human cells. We used CRISPR/Cas9 genome engineering of primary …
difficult to model in human cells. We used CRISPR/Cas9 genome engineering of primary …
CRISPR medicine for blood disorders: progress and challenges in delivery
T Mohammadian Gol, G Ureña-Bailén… - Frontiers in Genome …, 2023 - frontiersin.org
Blood disorders are a group of diseases including hematological neoplasms, clotting
disorders and orphan immune deficiency diseases that affects human health. Current …
disorders and orphan immune deficiency diseases that affects human health. Current …
CRISPR/Cas systems usher in a new era of disease treatment and diagnosis
R Li, Q Wang, K She, F Lu, Y Yang - Molecular Biomedicine, 2022 - Springer
The discovery and development of the CRISPR/Cas system is a milestone in precise
medicine. CRISPR/Cas nucleases, base-editing (BE) and prime-editing (PE) are three …
medicine. CRISPR/Cas nucleases, base-editing (BE) and prime-editing (PE) are three …
Gene editing and CRISPR in the clinic: current and future perspectives
Genome editing technologies, particularly those based on zinc-finger nucleases (ZFNs),
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …
Genome editing for human gene therapy
The rapid advancement of genome-editing techniques holds much promise for the field of
human gene therapy. From bacteria to model organisms and human cells, genome editing …
human gene therapy. From bacteria to model organisms and human cells, genome editing …
Therapeutic genome editing with engineered nucleases
SA Haas, V Dettmer, T Cathomen - Hämostaseologie, 2017 - thieme-connect.com
Targeted genome editing with designer nucleases, such as zinc finger nucleases, TALE
nucleases, and CRISPR-Cas nucleases, has heralded a new era in gene therapy. Genetic …
nucleases, and CRISPR-Cas nucleases, has heralded a new era in gene therapy. Genetic …
Current trends of clinical trials involving CRISPR/Cas systems
S Zhang, Y Wang, D Mao, Y Wang, H Zhang… - Frontiers in …, 2023 - frontiersin.org
The CRISPR/Cas9 system is a powerful genome editing tool that has made enormous
impacts on next-generation molecular diagnostics and therapeutics, especially for genetic …
impacts on next-generation molecular diagnostics and therapeutics, especially for genetic …
Genome and epigenome editing to treat disorders of the hematopoietic system
C Mussolino, J Alzubi, V Pennucci, G Turchiano… - Human gene …, 2017 - liebertpub.com
The possibility of editing complex genomes in a targeted fashion has revolutionized basic
research as well as biomedical and biotechnological applications in the last 5 years. The …
research as well as biomedical and biotechnological applications in the last 5 years. The …
Applying CRISPR-Cas9 screens to dissect hematological malignancies
DN Iyer, AD Schimmer, H Chang - Blood Advances, 2023 - ashpublications.org
Bit by bit, over the last few decades, functional genomic tools have been piecing together the
molecular puzzle driving tumorigenesis in human patients. Nevertheless, our understanding …
molecular puzzle driving tumorigenesis in human patients. Nevertheless, our understanding …