Results 81 to 90 of about 172,174 (293)

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

, 2012
The RPE65 gene encodes the isomerase of the retinoid cycle, the enzymatic pathway that underlies mammalian vision. Mutations in RPE65 disrupt the retinoid cycle and cause a congenital human blindness known as Leber congenital amaurosis (LCA).
Heon, Elise, Aleman, Tomas S., Cideciyan, Artur V., Roman, Alejandro J., Wilson, James M., Stone, Edwin M., Jacobson, Samuel G., Bryne, Barry J., Boye, Sanford L., Fishman, Gerald A., Windsor, Elizabeth A. M., Kaushal, Shalesh, Flotte, Terence R., Hauswirth, William W., Pang, Ji-Jing, Schwartz, Sharon B., Sumaroka, Alexander   +16 more
core   +1 more source

CCDC80 suppresses high‐grade serous ovarian cancer migration via negative regulation of B7‐H3

Molecular Oncology, EarlyView.
PAX8 is a lineage‐specific master regulator of transcription in high‐grade serous ovarian cancer (HGSC) progression. We show for the first time that PAX8 facilitates proliferation and metastasis by repressing the cell autonomous tumor suppressor CCDC80 and inducing B7‐H3 expression.
Aya Saleh, Nitzan Medina‐Itzhaki, Milena Chekov, Eden Gal‐Swisa, Roba Gabesh‐Wahabi, Inbar Savyon, Inna Naroditsky, Hilary A. Kenny, Basem Fares, Lina Korsensky, Einav Girsh, Liron Berger, Ruth Perets   +12 more
wiley   +1 more source

Heterozygous loss‐of‐function alleles associate the conserved 3′‐5′ exoribonuclease EXOSC10 with hypersensitivity to the anticancer drug 5‐fluorouracil

Molecular Oncology, EarlyView.
EXOSC10, an essential nuclear RNA exosome‐associated 3′‐5′ exoribonuclease, is inhibited by the anticancer drug 5‐fluorouracil (5‐FU), and EXOSC10 depletion increases 5‐FU sensitivity. The colon‐cancer variant EXOSC10S402T, located in a proteolysis motif, is stable and nuclear but nonfunctional in vivo.
Radhika Sain, Yann Le Page, Frédéric Percevault, Emmanuelle Becker, Luc Negroni, Almudena Fernandez, Marta Cantero, Diego Muñoz‐Santos, Lluís Montoliu, Cyrille Garnier, Michael Primig   +10 more
wiley   +1 more source

Oestrogen replacement therapy may improve memory functioning in the absence of APOE e4

, 2004
There is currently intense controversy regarding the use of hormone replacement therapy (HRT) in postmenopausal women, in relation to its therapeutic efficacy in Alzheimer's disease (AD).
Stuckey, B.G.A., Laws, S.M., Nolan, D., Hewson-Bower, B., Baker, L.D., Foster, J.K., Clarnette, R., Burkhardt, M.S., Martins, R.N., Gandy, S.E., Craft, S.   +10 more
core  

Chitosan and its modifications: are they possible vehicles for gene therapy [PDF]

, 2011
[Extract] In gene therapy, the most important step is how to effectively deliver the therapeutic gene to the target cells or organ. At present, there are two methods, which are those using a viral and a non-viral vector system.
Peter A. Leggat, Ureporn Kedjarune-Leggat, Leggat, Peter A., Peter A., Kedjarune-Leggat, Ureporn   +4 more
core   +1 more source

Transcriptional profiling of circulating extracellular vesicles from prebiopsy prostate cancer patients

Molecular Oncology, EarlyView.
RNA profiling of circulating extracellular vesicles (EVs) from blood samples of men undergoing prostate biopsy identifies transcripts associated with clinically significant prostate cancer. Integrative analysis with public tumor datasets links EV‐derived gene signatures to tumor stage and progression‐free survival, highlighting CASP3, XRCC2, and RIT1 ...
Stefan Werner, Pierre Tennstedt, Randi C. Pose, Christian Müller, Katharina Besler, Svenja Schneegans, Malik Alawi, Marie C. Roesch, Sven Peine, Desiree Bonci, Joanna Budna‐Tukan, Evi Lianidou, Catherine Alix‐Panabières, Derya Tilki, Klaus Pantel   +14 more
wiley   +1 more source

Diabetes gene therapy: potential and challenges.

, 2003
Gene therapy, developing rapidly as a result of advances in molecular biology and the Human Genome Project, is now highlighted as a most hopeful technology of the 21st century.
Lam, KS, Kung, HF, Li, H, Tse, LY, Xu, R, Lu, H   +5 more
core  

Recombinant Adeno-Associated Virus-Based Gene Therapy for Disorders Detected by Newborn Screening: Inherent Limitations of This Approach

, 2018
For more than 50 years, newborn screening (NBS) in the United States has been used for early detection of potentially lethal single gene disorders, such as phenylketonuria and congenital adrenal hyperplasia.1.
Flotte, Terence R.
core   +1 more source

GENE THERAPY: REPAIRING AND REPLACING DEFECTIVE GENES

As we navigate the complexities of the 21st century, the quest for sustainable solutions to global challenges has never been more urgent. "Harnessing Nature: Biotechnology in Medicine, Agriculture, and Beyond" explores the transformative power of biotechnology and its potential to address some of the most pressing issues facing humanity today.
Pandey, Ashutosh, Soni, Monika
openaire   +2 more sources

Interaction of HS1BP3 with cortactin modulates TKS5 localisation, cell secretion and cancer malignancy

Molecular Oncology, EarlyView.
Here, we demonstrate that HS1BP3 interacts with Cortactin through a proline‐rich region (PRR3.1) and show that this interaction, and HS1BP3 itself, promote cancer cell proliferation and invasion. Inhibition of this interaction leads to build‐up of TKS5 in multivesicular endosomes and altered secretion of CD63 and CD9, providing an explanation for the ...
Arja Arnesen Løchen, Kristiane Søreng, Chiara Veroni, Laura Trachsel‐Moncho, Nagham Asp, Robin Gaupset, Lars Gustav Lyckander, Helene Knævelsrud, Lars Eftang, Anne Simonsen   +9 more
wiley   +1 more source