[HTML][HTML] A rapid CRISPR competitive assay for in vitro and in vivo discovery of potential drug targets affecting the hematopoietic system

Y Shen, L Jiang, VS Iyer, B Raposo… - Computational and …, 2021 - Elsevier
CRISPR/Cas9 can be used as an experimental tool to inactivate genes in cells. However, a
CRISPR-targeted cell population will not show a uniform genotype of the targeted gene …

[HTML][HTML] Efficient CRISPR/Cas9-mediated gene knockin in mouse hematopoietic stem and progenitor cells

NT Tran, T Sommermann, R Graf, J Trombke, J Pempe… - Cell reports, 2019 - cell.com
Mutations accumulating in hematopoietic stem and progenitor cells (HSPCs) during
development can cause severe hematological disorders. Modeling these mutations in mice …

Genome Engineering of Hematopoietic Stem Cells Using CRISPR/Cas9 System

N Devaraju, V Rajendiran, NS Ravi… - Stem Cell Assays …, 2022 - Springer
Ex vivo genetic manipulation of autologous hematopoietic stem and progenitor cells
(HSPCs) is a viable strategy for the treatment of hematologic and primary immune disorders …

[HTML][HTML] Integration of CRISPR/Cas9 with artificial intelligence for improved cancer therapeutics

AA Bhat, S Nisar, S Mukherjee, N Saha… - Journal of translational …, 2022 - Springer
Gene editing has great potential in treating diseases caused by well-characterized
molecular alterations. The introduction of clustered regularly interspaced short palindromic …

[HTML][HTML] From Descriptive to Functional Genomics of Leukemias Focusing on Genome Engineering Techniques

B Balla, F Tripon, C Banescu - International Journal of Molecular Sciences, 2021 - mdpi.com
Genome engineering makes the precise manipulation of DNA sequences possible in a cell.
Therefore, it is essential for understanding gene function. Meganucleases were the start of …

[HTML][HTML] Whole-genome sequencing analysis reveals high specificity of CRISPR/Cas9 and TALEN-based genome editing in human iPSCs

C Smith, A Gore, W Yan, L Abalde-Atristain, Z Li, C He… - Cell stem cell, 2014 - cell.com
Human iPSCs provide renewable cell sources for human biology and disease research and
the potential for developing gene and cell therapy. Realization of this potential will rely in …

[HTML][HTML] Key challenges in bringing CRISPR-mediated somatic cell therapy into the clinic

D Nicol, L Eckstein, M Morrison, JS Sherkow… - Genome medicine, 2017 - Springer
Editorial summary Genome editing using clustered regularly interspersed short palindromic
repeats (CRISPR) and CRISPR-associated proteins offers the potential to facilitate safe and …

[HTML][HTML] CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations

G Cullot, J Boutin, J Toutain, F Prat… - Nature …, 2019 - nature.com
CRISPR-Cas9 is a promising technology for genome editing. Here we use Cas9 nuclease-
induced double-strand break DNA (DSB) at the UROS locus to model and correct congenital …

[HTML][HTML] Programmable molecular scissors: applications of a new tool for genome editing in biotech

SK Saha, FK Saikot, MS Rahman, MAHM Jamal… - … Therapy-Nucleic Acids, 2019 - cell.com
Targeted genome editing is an advanced technique that enables precise modification of the
nucleic acid sequences in a genome. Genome editing is typically performed using tools …

[HTML][HTML] COSMID: a web-based tool for identifying and validating CRISPR/Cas off-target sites

TJ Cradick, P Qiu, CM Lee, EJ Fine, G Bao - Molecular Therapy-Nucleic …, 2014 - cell.com
Precise genome editing using engineered nucleases can significantly facilitate biological
studies and disease treatment. In particular, clustered regularly interspaced short …