Results 41 to 50 of about 12,985,653 (406)

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

open access: yes, 2004
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Braun-Falco, M., Buning, H., Hallek, M.
core   +1 more source

Gene targeting, genome editing: from Dolly to editors

open access: yesTransgenic research, 2016
One of the most powerful strategies to investigate biology we have as scientists, is the ability to transfer genetic material in a controlled and deliberate manner between organisms.
W. Tan   +3 more
semanticscholar   +1 more source

Molecular bases of low production rates of apolipoprotein B-100 and truncated apoB-82 in a mutant HepG2 cell line generated by targeted modification of the apolipoprotein B gene

open access: yesJournal of Lipid Research, 1999
In subjects with familial hypobetalipoproteinemia heterozygous for truncated forms of apolipoprotein B, both apoB-100 and the truncated forms are produced at lower than expected rates.
Rai Ajit K. Srivastava   +4 more
doaj   +1 more source

Targeted genome modifications in soybean with CRISPR/Cas9 [PDF]

open access: yes, 2015
Background: The ability to selectively alter genomic DNA sequences in vivo is a powerful tool for basic and applied research. The CRISPR/Cas9 system precisely mutates DNA sequences in a number of organisms.
Jacobs, Thomas   +3 more
core   +2 more sources

Practical guidance for the implementation of the CRISPR genome editing tool in filamentous fungi [PDF]

open access: yes, 2019
Background: Within the last years, numerous reports described successful application of the CRISPR nucleases Cas9 and Cpf1 for genome editing in filamentous fungi.
Haefner, Stefan   +4 more
core   +1 more source

A PCA3 gene-based transcriptional amplification system targeting primary prostate cancer. [PDF]

open access: yes, 2015
Targeting specifically primary prostate cancer (PCa) cells for immune therapy, gene therapy or molecular imaging is of high importance. The PCA3 long non-coding RNA is a unique PCa biomarker and oncogene that has been widely studied.
Fradet, Yves   +5 more
core   +2 more sources

Targeting the Apoa1 locus for liver-directed gene therapy

open access: yesMolecular Therapy: Methods & Clinical Development, 2021
Clinical application of somatic genome editing requires therapeutics that are generalizable to a broad range of patients. Targeted insertion of promoterless transgenes can ensure that edits are permanent and broadly applicable while minimizing risks of ...
Marco De Giorgi   +11 more
doaj   +1 more source

Life cycle studies of the hexose transporter of Plasmodium species and genetic validation of their essentiality [PDF]

open access: yes, 2010
A Plasmodium falciparum hexose transporter (PfHT) has previously been shown to be a facilitative glucose and fructose transporter. Its expression in Xenopus laevis oocytes and the use of a glucose analogue inhibitor permitted chemical validation of PfHT ...
Doerig, C   +6 more
core   +2 more sources

High-throughput gene targeting and phenotyping in zebrafish using CRISPR/Cas9

open access: yesGenome Research, 2015
The use of CRISPR/Cas9 as a genome-editing tool in various model organisms has radically changed targeted mutagenesis. Here, we present a high-throughput targeted mutagenesis pipeline using CRISPR/Cas9 technology in zebrafish that will make possible both
G. Varshney   +16 more
semanticscholar   +1 more source

Leishmania infantum Lipophosphoglycan-Deficient Mutants: A Tool to Study Host Cell-Parasite Interplay

open access: yesFrontiers in Microbiology, 2018
Lipophosphoglycan (LPG) is the major surface glycoconjugate of metacyclic Leishmania promastigotes and is associated with virulence in various species of this parasite.
Milena Lázaro-Souza   +18 more
doaj   +1 more source

Home - About - Disclaimer - Privacy