Results 101 to 110 of about 1,008,568 (216)

LATEST ADVANCES FOR TREATING CONGENITAL ADRENAL HYPERPLASIA DUE TO 21-HYDROXYLASE DEFICIENCY

İstanbul Tıp Fakültesi Dergisi
Congenital adrenal hyperplasia (CAH) is a group of inherit ed diseases characterised by disrupted glucocorticoid (GC) and mineralocorticoid (MC) synthesis in the adrenal glands.
Ecesu Çetin, Mehmet Furkan Burak
doaj   +1 more source

Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models. [PDF]

Gene Ther, 2022
Chilcott EM, Muiruri EW, Hirst TC, Yáñez-Muñoz RJ.   +3 more
europepmc   +1 more source

Extended Toxicity, Genotoxicity, and Mutagenicity of Combination of pBudK-coVEGF-coANG and pBudK-coGDNF Plasmids in Preclinical Trials

Biomedicines
Chronic lower limb ischemia is a debilitating condition, particularly prevalent among elderly patients and individuals ineligible for revascularization procedures.
Igor V. Samatoshenkov, Alexander M. Aimaletdinov, Elena Y. Zakirova, Egan L. Kalmykov, Rustam Khodzhibaev, Yulia M. Samatoshenkova, Ilnur M. Ganiev, Marat S. Kadyrov, Yana O. Mukhamedshina   +8 more
doaj   +1 more source

Adenine base editing of the DUX4 polyadenylation signal for targeted genetic therapy in facioscapulohumeral muscular dystrophy. [PDF]

Mol Ther Nucleic Acids, 2021
Šikrová D, Cadar VA, Ariyurek Y, Laros JFJ, Balog J, van der Maarel SM.   +5 more
europepmc   +1 more source

Extravascular factor IX after gene therapy in hemophilia B, does it matter?

Research and Practice in Thrombosis and Haemostasis
Gene therapy will very likely change the treatment paradigm of hemophilia B in the coming years. For the majority of patients, adjunctive exogenous factor (F)IX clotting factor concentrate will continue to be needed in case of surgery or bleeding ...
Quentin Van Thillo, Cédric Hermans
doaj   +1 more source

With newer tools for gene editing, is it time to revisit genetic therapy for cystic fibrosis? [PDF]

Genes Dis, 2021
Gupta A, Gupta A, Boyd L.
europepmc   +1 more source

Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I. [PDF]

Front Mol Neurosci, 2021
Belur LR, Romero M, Lee J, Podetz-Pedersen KM, Nan Z, Riedl MS, Vulchanova L, Kitto KF, Fairbanks CA, Kozarsky KF, Orchard PJ, Frey WH, Low WC, McIvor RS.   +13 more
europepmc   +1 more source

Biosafety Evaluation of a Chimeric Adenoviral Vector in Mini-Pigs: Insights into Immune Tolerance and Gene Therapy Potential

Biomedicines
Background: The biosafety of gene therapy products remains a major challenge to their introduction into the clinic. In particular, the problem of immunogenicity of viral vectors is the focus of attention.
Andrei Izmailov, Irina Minyazeva, Vage Markosyan, Zufar Safiullov, Ilnaz Gazizov, Ilnur Salafutdinov, Maria Markelova, Ravil Garifulin, Maksim Shmarov, Denis Logunov, Rustem Islamov, Vadim Pospelov   +11 more
doaj   +1 more source

Genetic and immunological therapy for cancer [PDF]

Journal of the Royal Society of Medicine, 2000
A, Young, D J, Kerr
openaire   +2 more sources

Therapeutic future of Fuchs endothelial corneal dystrophy: An ongoing way to explore

Taiwan Journal of Ophthalmology
Fuchs endothelial corneal dystrophy (FECD) is one of the most common corneal diseases that causes loss of visual acuity in the world. FECD is a genetically and pathogenetically heterogeneous disease that results in the failure of corneal endothelial ...
Jia-Xin Liu, Tung-Lin Chiang, Kai-Feng Hung, Yi-Chen Sun   +3 more
doaj   +1 more source