Results 111 to 120 of about 708,347 (229)
Targeted gene repair – in the arena [PDF]
Journal of Clinical Investigation, 2003 The development of targeted gene repair is under way and, despite some setbacks, shows promise as an alternative form of gene therapy. This approach uses synthetic DNA molecules to activate and direct the cell's inherent DNA repair systems to correct inborn errors.
openaire +3 more sources
Targeting the Apoa1 locus for liver-directed gene therapy
Molecular 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
Prediction of a Gene Regulatory Network from Gene Expression Profiles With Linear Regression and Pearson Correlation Coefficient [PDF]
arXiv, 2018 Reconstruction of gene regulatory networks is the process of identifying gene dependency from gene expression profile through some computation techniques. In our human body, though all cells pose similar genetic material but the activation state may vary.
arxiv
Molecular Therapy: Methods & Clinical Development, 2020 Cystic fibrosis (CF) is the main genetic cause of death among the Caucasian population. The disease is characterized by abnormal fluid and electrolyte mobility across secretory epithelia.
Aarne Fleischer +12 more
doaj
CRISPR/Cascade 9-Mediated Genome Editing-Challenges and Opportunities
Frontiers in Genetics, 2018 Clustered Regularly Interspaced Palindromic Repeats (CRISPR) and Cascade 9 (also known as Cas9, CRISPR associated protein 9) confer protection against invading viruses or plasmids.
Bhaskar Roy +15 more
doaj +1 more source
Viruses, 2015 An engineered RNase P-based ribozyme variant, which was generated using the in vitro selection procedure, was used to target the overlapping mRNA region of two proteins essential for human cytomegalovirus (HCMV) replication: capsid assembly protein (AP ...
Zhu Yang +9 more
doaj +1 more source
Biomolecules, 2019 CRISPR/Cas9 is a powerful tool for genome editing in cells and organisms. Nevertheless, introducing directed templated changes by homology-directed repair (HDR) requires the cellular DNA repair machinery, such as the MRN complex (Mre11/Rad50/Nbs1).
Nina Reuven +4 more
doaj +1 more source
Targeted and nontargeted liposomes for in vivo transfer to rat liver cells of a plasmid containing the preproinsulin I gene. [PDF]
, 1983 Philippe Soriano +7 more
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Gene Therapy: The Potential Applicability of Gene Transfer Technology to the Human Germline
International Journal of Medical Sciences, 2004 The theoretical possibility of applying gene transfer methodologies to the human germline is explored. Transgenic methods for genetically manipulating embryos may in principle be applied to humans.
doaj
Two target sites for protein binding in the promoter region of a cell cycle regulated human H1 histone gene [PDF]
, 1988 André J. van Wijnen +5 more
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