[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 …

CRISPR technologies and the search for the PAM-free nuclease

D Collias, CL Beisel - Nature communications, 2021 - nature.com
The ever-expanding set of CRISPR technologies and their programmable RNA-guided
nucleases exhibit remarkable flexibility in DNA targeting. However, this flexibility comes with …

The APOE-R136S mutation protects against APOE4-driven Tau pathology, neurodegeneration and neuroinflammation

MR Nelson, P Liu, A Agrawal, O Yip, J Blumenfeld… - Nature …, 2023 - nature.com
Abstract Apolipoprotein E4 (APOE4) is the strongest genetic risk factor for late-onset
Alzheimer's disease (LOAD), leading to earlier age of clinical onset and exacerbating …

Generation of human striatal organoids and cortico-striatal assembloids from human pluripotent stem cells

Y Miura, MY Li, F Birey, K Ikeda, O Revah… - Nature …, 2020 - nature.com
Cortico-striatal projections are critical components of forebrain circuitry that regulate
motivated behaviors. To enable the study of the human cortico-striatal pathway and how its …

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

CA Vakulskas, DP Dever, GR Rettig, R Turk… - Nature medicine, 2018 - nature.com
Translation of the CRISPR–Cas9 system to human therapeutics holds high promise.
However, specificity remains a concern especially when modifying stem cell populations …

CRISPR/Cas9 mediated deletion of the adenosine A2A receptor enhances CAR T cell efficacy

L Giuffrida, K Sek, MA Henderson, J Lai… - Nature …, 2021 - nature.com
Adenosine is an immunosuppressive factor that limits anti-tumor immunity through the
suppression of multiple immune subsets including T cells via activation of the adenosine …

Efficient introduction of specific homozygous and heterozygous mutations using CRISPR/Cas9

D Paquet, D Kwart, A Chen, A Sproul, S Jacob, S Teo… - Nature, 2016 - nature.com
The bacterial CRISPR/Cas9 system allows sequence-specific gene editing in many
organisms and holds promise as a tool to generate models of human diseases, for example …

CRISPR/Cas9-based genome editing for disease modeling and therapy: challenges and opportunities for nonviral delivery

HX Wang, M Li, CM Lee, S Chakraborty… - Chemical …, 2017 - ACS Publications
Genome editing offers promising solutions to genetic disorders by editing DNA sequences
or modulating gene expression. The clustered regularly interspaced short palindromic …

CD7-edited T cells expressing a CD7-specific CAR for the therapy of T-cell malignancies

D Gomes-Silva, M Srinivasan, S Sharma… - Blood, The Journal …, 2017 - ashpublications.org
Extending the success of chimeric antigen receptor (CAR) T cells to T-cell malignancies is
problematic because most target antigens are shared between normal and malignant cells …

Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

A Lattanzi, J Camarena, P Lahiri, H Segal… - Science translational …, 2021 - science.org
Sickle cell disease (SCD) is the most common serious monogenic disease with 300,000
births annually worldwide. SCD is an autosomal recessive disease resulting from a single …