Results 51 to 60 of about 1,207,125 (312)

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, Ang Li, Ayrea Hurley, Mercedes Barzi, Alexandria M. Doerfler, Nikitha A. Cherayil, Harrison E. Smith, Jonathan D. Brown, Charles Y. Lin, Karl-Dimiter Bissig, Gang Bao, William R. Lagor   +11 more
doaj   +1 more source

Organoids in pediatric cancer research

FEBS Letters, EarlyView.
Organoid technology has revolutionized cancer research, yet its application in pediatric oncology remains limited. Recent advances have enabled the development of pediatric tumor organoids, offering new insights into disease biology, treatment response, and interactions with the tumor microenvironment.
Carla Ríos Arceo, Jarno Drost
wiley   +1 more source

Osteoblast-Targeting-Peptide Modified Nanoparticle for siRNA/microRNA Delivery [PDF]

, 2016
Antiosteoporosis gene-based drug development strategies are presently focused on targeting osteoblasts to either suppress bone loss or increase bone mass.
Xiangning Liu (431172), An Hong (2747227), Xiongzhen Ye (2747224), Bin Zhang (64682), Yayu Wang (1658227), Jia Xiao (553000), Jiafan Liu (2747230), Xiaogang Wang (250803), Mingxiang Cai (282695), Zuolin Wang (2747233), Yao Sun (145327), Chen Kang (1416142), Chenyang Zhang (1383045), Li Yang (6520), Qi Zhang (28502), Shannai Li (2747236)   +15 more
core   +1 more source

Hepatic secretion of apoB-100 is impaired in hypobetalipoproteinemic mice with an apoB-38.9-specifying allele

Journal of Lipid Research, 2004
Apolipoprotein B (apoB) truncation-specifying mutations cause familial hypobetalipoproteinemia (FHBL). Lipoprotein kinetics studies have shown that production rates of apoB-100 are reduced by 70–80% in heterozygous FHBL humans, instead of the expected 50%
Zhouji Chen, Robin L. Fitzgerald, Gang Li, Nicholas O. Davidson, Gustav Schonfeld   +4 more
doaj   +1 more source

Precise Genome Editing in miRNA Target Site via Gene Targeting and Subsequent Single-Strand-Annealing-Mediated Excision of the Marker Gene in Plants

Frontiers in Genome Editing, 2021
Gene targeting (GT) enables precise genome modification—e.g., the introduction of base substitutions—using donor DNA as a template. Combined with clean excision of the selection marker used to select GT cells, GT is expected to become a standard ...
Namie Ohtsuki, Keiko Kizawa, Akiko Mori, Ayako Nishizawa-Yokoi, Ayako Nishizawa-Yokoi, Takao Komatsuda, Hitoshi Yoshida, Katsuyuki Hayakawa, Seiichi Toki, Seiichi Toki, Seiichi Toki, Hiroaki Saika   +11 more
doaj   +1 more source

Reciprocal control of viral infection and phosphoinositide dynamics

FEBS Letters, EarlyView.
Phosphoinositides, although scarce, regulate key cellular processes, including membrane dynamics and signaling. Viruses exploit these lipids to support their entry, replication, assembly, and egress. The central role of phosphoinositides in infection highlights phosphoinositide metabolism as a promising antiviral target.
Marie Déborah Bancilhon, Bruno Mesmin
wiley   +1 more source

Targeting the MLL complex in acute leukemia [PDF]

, 2014
Chromosomal rearrangements leading mostly to fusion oncoproteins of the Mixed Lineage Leukemia (MLL) gene occur in about 10% of all patients with acute leukemia and are often associated with poor clinical outcome, emphasizing the need for new treatment ...
Méreau, Hélène
core   +1 more source

The Gene Targeting Approach of Small Fragment Homologous Replacement (SFHR) Alters the Expression Patterns of DNA Repair and Cell Cycle Control Genes

Molecular Therapy: Nucleic Acids, 2016
Cellular responses and molecular mechanisms activated by exogenous DNA that invades cells are only partially understood. This limits the practical use of gene targeting strategies.
Silvia Pierandrei, Andrea Luchetti, Massimo Sanchez, Giuseppe Novelli, Federica Sangiuolo, Marco Lucarelli   +5 more
doaj   +1 more source

Phosphatidylinositol 4‐kinase as a target of pathogens—friend or foe?

FEBS Letters, EarlyView.
This graphical summary illustrates the roles of phosphatidylinositol 4‐kinases (PI4Ks). PI4Ks regulate key cellular processes and can be hijacked by pathogens, such as viruses, bacteria and parasites, to support their intracellular replication. Their dual role as essential host enzymes and pathogen cofactors makes them promising drug targets.
Ana C. Mendes, Guilherme M. Azevedo, Amanda P. Barcellos, Diana Bahia   +3 more
wiley   +1 more source

Targeting and dynamics of gene repression during stem cell differentiation [PDF]

, 2011
The identity and function of different cellular subtypes critically depend on their unique set of expressed genes. Gene expression programs and their changes during development are mainly controlled by sequence-specific DNA binding factors.
Lienert, Florian
core   +1 more source