Viral Vectors for the in Vivo Delivery of CRISPR Components: Advances and Challenges
M Asmamaw Mengstie - Frontiers in Bioengineering and …, 2022 - frontiersin.org
The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) and its
accompanying protein (Cas9) are now the most effective, efficient, and precise genome …
accompanying protein (Cas9) are now the most effective, efficient, and precise genome …
Immunogenicity of CRISPR therapeutics—Critical considerations for clinical translation
R Ewaisha, KS Anderson - Frontiers in Bioengineering and …, 2023 - frontiersin.org
CRISPR offers new hope for many patients and promises to transform the way we think of
future therapies. Ensuring safety of CRISPR therapeutics is a top priority for clinical …
future therapies. Ensuring safety of CRISPR therapeutics is a top priority for clinical …
Programmable RNA editing with compact CRISPR–Cas13 systems from uncultivated microbes
C Xu, Y Zhou, Q Xiao, B He, G Geng, Z Wang, B Cao… - Nature …, 2021 - nature.com
Competitive coevolution between microbes and viruses has led to the diversification of
CRISPR–Cas defense systems against infectious agents. By analyzing metagenomic …
CRISPR–Cas defense systems against infectious agents. By analyzing metagenomic …
[HTML][HTML] Development of CRISPR as an antiviral strategy to combat SARS-CoV-2 and influenza
Summary The coronavirus disease 2019 (COVID-19) pandemic, caused by the SARS-CoV-
2 virus, has highlighted the need for antiviral approaches that can target emerging viruses …
2 virus, has highlighted the need for antiviral approaches that can target emerging viruses …
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 …
such as gene therapy or bispecific antibody therapy have been introduced, substantial …
Generation of precision preclinical cancer models using regulated in vivo base editing
Although single-nucleotide variants (SNVs) make up the majority of cancer-associated
genetic changes and have been comprehensively catalogued, little is known about their …
genetic changes and have been comprehensively catalogued, little is known about their …
Detect and destroy: CRISPR-based technologies for the response against viruses
CA Freije, PC Sabeti - Cell host & microbe, 2021 - cell.com
Despite numerous viral outbreaks in the last decade, including a devastating global
pandemic, diagnostic and therapeutic technologies remain severely lacking. CRISPR-Cas …
pandemic, diagnostic and therapeutic technologies remain severely lacking. CRISPR-Cas …
Gene therapy: a double-edged sword with great powers
R Tang, Z Xu - Molecular and cellular biochemistry, 2020 - Springer
Gene therapy is the treatment of a disease through transferring genetic material into cells of
the patients. In the recent several years, gene therapy has experienced rapid progress and …
the patients. In the recent several years, gene therapy has experienced rapid progress and …
Control of mammalian gene expression by modulation of polyA signal cleavage at 5′ UTR
L Luo, JDY Jea, Y Wang, PW Chao, L Yen - Nature biotechnology, 2024 - nature.com
The ability to control gene expression in mammalian cells is crucial for safe and efficacious
gene therapies and for elucidating gene functions. Current gene regulation systems have …
gene therapies and for elucidating gene functions. Current gene regulation systems have …
Correction of muscular dystrophies by CRISPR gene editing
F Chemello, R Bassel-Duby… - The Journal of clinical …, 2020 - Am Soc Clin Investig
Muscular dystrophies are debilitating disorders that result in progressive weakness and
degeneration of skeletal muscle. Although the genetic mutations and clinical abnormalities …
degeneration of skeletal muscle. Although the genetic mutations and clinical abnormalities …