Off-target effects in CRISPR/Cas9 gene editing

C Guo, X Ma, F Gao, Y Guo - Frontiers in bioengineering and …, 2023 - frontiersin.org
Gene editing stands for the methods to precisely make changes to a specific nucleic acid
sequence. With the recent development of the clustered regularly interspaced short …

Advances in CRISPR therapeutics

M Chavez, X Chen, PB Finn, LS Qi - Nature Reviews Nephrology, 2023 - nature.com
The clustered regularly interspaced short palindromic repeats (CRISPR) renaissance was
catalysed by the discovery that RNA-guided prokaryotic CRISPR-associated (Cas) proteins …

Mechanism and applications of CRISPR/Cas-9-mediated genome editing

M Asmamaw, B Zawdie - Biologics: targets and therapy, 2021 - Taylor & Francis
Clustered regularly interspaced short palindromic repeat (CRISPR) and their associated
protein (Cas-9) is the most effective, efficient, and accurate method of genome editing tool in …

[HTML][HTML] Massively parallel base editing to map variant effects in human hematopoiesis

JD Martin-Rufino, N Castano, M Pang, EI Grody… - Cell, 2023 - cell.com
Systematic evaluation of the impact of genetic variants is critical for the study and treatment
of human physiology and disease. While specific mutations can be introduced by genome …

Stimuli-responsive gene delivery nanocarriers for cancer therapy

Q Zhang, G Kuang, W Li, J Wang, H Ren, Y Zhao - Nano-Micro Letters, 2023 - Springer
Gene therapy provides a promising approach in treating cancers with high efficacy and
selectivity and few adverse effects. Currently, the development of functional vectors with …

In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy

JP Han, MJ Kim, BS Choi, JH Lee, GS Lee, M Jeong… - Science …, 2022 - science.org
Hemophilia is a hereditary disease that remains incurable. Although innovative treatments
such as gene therapy or bispecific antibody therapy have been introduced, substantial …

Current state of human gene therapy: approved products and vectors

AY Shchaslyvyi, SV Antonenko, MG Tesliuk… - Pharmaceuticals, 2023 - mdpi.com
In the realm of gene therapy, a pivotal moment arrived with Paul Berg's groundbreaking
identification of the first recombinant DNA in 1972. This achievement set the stage for future …

[HTML][HTML] Current and future concepts for the generation and application of genetically engineered CAR-T and TCR-T cells

M Hiltensperger, AM Krackhardt - Frontiers in Immunology, 2023 - frontiersin.org
Adoptive cell therapy (ACT) has seen a steep rise of new therapeutic approaches in its
immune-oncology pipeline over the last years. This is in great part due to the recent …

Gene therapy for CNS disorders: modalities, delivery and translational challenges

J Gao, S Gunasekar, Z **a, K Shalin, C Jiang… - Nature Reviews …, 2024 - nature.com
Gene therapy is emerging as a powerful tool to modulate abnormal gene expression, a
hallmark of most CNS disorders. The transformative potentials of recently approved gene …

[HTML][HTML] A critical review: recent advancements in the use of CRISPR/Cas9 technology to enhance crops and alleviate global food crises

A Rasheed, RA Gill, MU Hassan, A Mahmood… - Current Issues in …, 2021 - mdpi.com
Genome editing (GE) has revolutionized the biological sciences by creating a novel
approach for manipulating the genomes of living organisms. Many tools have been …