Results 1 to 10 of about 1,003,750 (315)

Genetic Therapy Approaches for Ornithine Transcarbamylase Deficiency [PDF]

Biomedicines, 2023
Ornithine transcarbamylase deficiency (OTCD) is the most common urea cycle disorder with high unmet needs, as current dietary and medical treatments may not be sufficient to prevent hyperammonemic episodes, which can cause death or neurological sequelae.
Berna Seker Yilmaz, Paul Gissen
core   +2 more sources

Tailoring therapy for heart failure: the pharmacogenomics of adrenergic receptor signaling

, 2014
Grazia Daniela Femminella,1 Vincenzo Barrese,2,3 Nicola Ferrara,1,4 Giuseppe Rengo4 1Department of Translational Medical Sciences, Federico II University, Naples, Italy; 2Department of Neuroscience, Reproductive Science and Odontostomatology, Federico II
Ferrara, N, Barrese V, Ferrara N, FERRARA, NICOLA, Rengo, G, Barrese, Vincenzo, FEMMINELLA, GRAZIA DANIELA, Femminella GD, Barrese, V, Rengo G, Femminella, GD, RENGO, GIUSEPPE   +11 more
core   +1 more source

Assessment of the frequency and association with morbidity of DNA markers in multinational administrative divisions based on indigenous population data (based on cardiovascular diseases)

Кардиоваскулярная терапия и профилактика
Information on morbidity is presented in statistical reports for the entire population of multinational subjects of Russia, but population Biobanks contain information on individual peoples.Aim.
E. V. Balanovskaya, A. Yu. Potanina, S. M. Koshel, D. S. Adamov, I. V. Evsyukov, A. L. Borisova, M. S. Pokrovskaya, A. N. Meshkov   +7 more
doaj   +1 more source

Cystic Fibrosis: New Trends in Therapy Methods

Архивъ внутренней медицины
This review provides information on recent advancements in the treatment of cystic fi brosis and presents interim results from ongoing clinical trials. Various scientifi c databases, including Scopus, Web of Science, and EMBASE, were utilized during the ...
P. A. Suchkova, S. A. Panova, O. Ya. Lisenko, K. P. Raevskij   +3 more
doaj   +1 more source

Advances in cell transplantation therapy and gene therapy in Parkinson's disease

Chinese Journal of Contemporary Neurology and Neurosurgery, 2022
The role of cell transplantation therapy and gene therapy in the treatment of Parkinson's disease (PD) has attracted more and more attention. It has developed from laboratory research tools to clinical products for patients.
XU Bao⁃lei, CHAN Piu
doaj   +1 more source

A theological consideration of ethical issues raised by human genetic manipulation, with particular reference to gene therapy [PDF]

, 1999
Human genetic manipulation is considered in terms of genetic testing and screening, gene therapy and enhancement, and reproductive cloning. Deontological and utilitarian approaches to ethical decision-making prove less than satisfactory.
Elkington, Audrey Anne
core  

AON-based degradation of c.151C>T mutant COCH transcripts associated with dominantly inherited hearing impairment DFNA9

Molecular Therapy: Nucleic Acids, 2021
The c.151C>T founder mutation in COCH is a frequent cause of late-onset, dominantly inherited hearing impairment and vestibular dysfunction (DFNA9) in the Dutch/Belgian population.
Erik de Vrieze, Jorge Cañas Martín, Jolien Peijnenborg, Aniek Martens, Jaap Oostrik, Simone van den Heuvel, Kornelia Neveling, Ronald Pennings, Hannie Kremer, Erwin van Wijk   +9 more
doaj   +1 more source

Pharmacogenetics of Migraine: genetic variants and their potential role in migraine therapy

, 2007
Migraine is a paroxysmal neurological disorder affecting up to 6% of males and 18% of females in the general population, and has been demonstrated to have a strong, but complex, genetic component.
LR Griffiths, Fernandez, F, Fernandez, Francesca, Griffiths, Lyn R., Griffiths, LR, Colson, Natalie, Colson, N. J., Griffiths, Lyn, NJ Colson, F Fernandez, Colson, NJ   +10 more
core   +1 more source

Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice

, 2022
α1-antitrypsin deficiency is a rare genetic condition that can cause liver and/or lung disease. There is currently no cure for this disorder, although repeated infusions of plasma-purified protein may slow down emphysema progression.
Zieger, Marina, Greer, Cynthia, Flotte, Terence R, Mueller, Christian, Borel, Florie, Blackwood, Meghan, Gernoux, Gwladys   +6 more
core   +1 more source

Optimizing milk-derived exosome carriers through systematic cationic material screening for efficient pulmonary-targeted mRNA delivery

陆军军医大学学报
Objective To construct an engineered milk-derived exosome (mExos) vector for efficient messenger RNA (mRNA) delivery to the respiratory tract by systematically screening cationic modification materials.
LUO Mingxing, LIAO Rui
doaj   +1 more source