Results 11 to 20 of about 60,004 (331)
Granulin knock out zebrafish lack frontotemporal lobar degeneration and neuronal ceroid lipofuscinosis pathology. [PDF]
PLoS ONE, 2015 Loss of function mutations in granulin (GRN) are linked to two distinct neurological disorders, frontotemporal lobar degeneration (FTLD) and neuronal ceroid lipofuscinosis (NCL).
Barbara Solchenberger +4 more
doaj +8 more sources
Frontiers in Bioengineering and Biotechnology, 2023 High-throughput genetic screening is frequently employed to rapidly associate gene with phenotype and establish sequence-function relationships. With the advent of CRISPR technology, and the ability to functionally interrogate previously genetically ...
Lisa Simirenko +4 more
doaj +5 more sources
CRISPRon/off: CRISPR/Cas9 on- and off-target gRNA design [PDF]
Bioinformatics, 2022 Summary The effectiveness of CRISPR/Cas9-mediated genome editing experiments largely depends on the guide RNA (gRNA) used by the CRISPR/Cas9 system for target recognition and cleavage activation.
Christian Anthon, G. Corsi, J. Gorodkin
semanticscholar +4 more sources
Synthetic gRNA/Cas9 Ribonucleoprotein Inhibits HIV Reactivation and Replication
Viruses, 2022 The current antiretroviral therapy (ART) for human immunodeficiency virus (HIV) can halt viral replication but cannot eradicate HIV infection because proviral DNA integrated into the host genome remains genetically silent in reservoir cells and is ...
Sushant Khanal +14 more
semanticscholar +4 more sources
Structure-based design of gRNA for Cas13 [PDF]
Scientific Reports, 2020 Cas13 endonuclease activity depends on the RNA local secondary structure with strong preference for single-stranded (SS) regions. Hence, it becomes indispensable to identify the SS regions for effective Cas13 mediated RNA knockdown.
S. Bandaru +9 more
semanticscholar +3 more sources
Scientific Reports, 2019 CRISPR-Cas9/gRNA exhibits therapeutic efficacy against latent human immunodeficiency virus (HIV) genome but the delivery of this therapeutic cargo to the brain remains as a challenge.
Ajeet K Kaushik +2 more
exaly +2 more sources
Nucleic Acids Research, 2020 A key aim in exploiting CRISPR-Cas is the engineering of gRNA to introduce additional functionalities, ranging from small nucleotide changes that increase efficiency of on-target binding to the inclusion of large functional RNA aptamers and ...
Grace Mullally +2 more
exaly +2 more sources
CRISPR-Cas9 Dual-gRNA Attack Causes Mutation, Excision and Inversion of the HIV-1 Proviral DNA
Viruses, 2020 Although several studies demonstrated that the HIV proviral DNA can be effectively targeted and inactivated by the CRISPR-Cas9 system, the precise inactivation mechanism has not yet been analyzed.
Caroline S. Binda +3 more
doaj +2 more sources
Efficient genome editing using tRNA promoter-driven CRISPR/Cas9 gRNA in Aspergillus niger. [PDF]
PLoS ONE, 2018 As a powerful tool for fast and precise genome editing, the CRISPR/Cas9 system has been applied in filamentous fungi to improve the efficiency of genome alteration.
Letian Song +4 more
doaj +2 more sources
Nature Communications, 2019 Understanding the impact of guide RNA (gRNA) and genomic locus on CRISPR-Cas9 activity is crucial to design effective gene editing assays. However, it is challenging to profile Cas9 activity in the endogenous cellular environment.
Santiago Gisler +2 more
exaly +2 more sources