المؤلفون: Ellethy, Abousree T. M., Hassan, Gamal M. A.

المصدر: International Journal of Recent Advances in Multidisciplinary Topics; Vol. 5 No. 4 (2024); 65-72 ; 2582-7839

مصطلحات موضوعية: gene therapy, monogenic and polygenic disorders, CRISPR-Cas9, genome editing delivery system, recombinant adeno-associated virus vectors, exosome, genetic disorders, liver diseases, hemophilia, FDA

وصف الملف: application/pdf

العلاقة: https://journals.ijramt.com/index.php/ijramt/article/view/2889/2905Test; https://journals.ijramt.com/index.php/ijramt/article/view/2889Test

الإتاحة: https://doi.org/10.5281/zenodo.11067723Test
https://journals.ijramt.com/index.php/ijramt/article/view/2889Test

المؤلفون: John K. Hall, Farid Moussavi-Harami, Stephen C. Kolwicz, Jeffrey S. Chamberlain, Stephen D. Hauschka, Xiolan Chen, Michael Regnier, Guy L. Odom

المصدر: JACC: Basic to Translational Science, Vol 4, Iss 7, Pp 778-791 (2019)
JACC: Basic to Translational Science

مصطلحات موضوعية: 0301 basic medicine, musculoskeletal diseases, medicine.medical_specialty, congenital, hereditary, and neonatal diseases and abnormalities, lcsh:Diseases of the circulatory (Cardiovascular) system, Duchenne muscular dystrophy, Genetic enhancement, viruses, Cardiomyopathy, rAAV, recombinant adeno-associated viral vector, 030204 cardiovascular system & hematology, ribonucleotide reductase, dystrophin, 03 medical and health sciences, PRECLINICAL RESEARCH, 0302 clinical medicine, μDys, microdystrophin, Internal medicine, recombinant adeno-associated virus vectors, cTnT, cardiac troponin T, Medicine, Vector (molecular biology), biology, business.industry, dADP, deoxy-adenosine diphosphate, Cardiac muscle, Cardiac reserve, RNR, ribonucleotide reductase, CMV, cytomegalovirus, DMD, Duchenne muscular dystrophy, medicine.disease, 3. Good health, 030104 developmental biology, Endocrinology, medicine.anatomical_structure, Ribonucleotide reductase, lcsh:RC666-701, CK8, miniaturized murine creatine kinase regulatory cassette, biology.protein, diastolic dysfunction, dATP, deoxy-adenosine triphosphate, Cardiology and Cardiovascular Medicine, business, Dystrophin, cardiomyopathy, mdx, mouse muscular dystrophy model

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::35da563ba80c05668887e4f5b7413550Test
http://www.sciencedirect.com/science/article/pii/S2452302X19301913Test

المؤلفون: Daniel J. Garry, Tamas Alexy

المصدر: JACC: Basic to Translational Science

مصطلحات موضوعية: biology, business.industry, Cardiomyopathy, ribonucleotide reductase, medicine.disease, dystrophin, Dystrophic cardiomyopathy, Ribonucleotide reductase, recombinant adeno-associated virus vectors, Cancer research, biology.protein, Medicine, diastolic dysfunction, Cardiology and Cardiovascular Medicine, business, Dystrophin, Editorial Comment, cardiomyopathy

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::26314fd9f99629c544ced9949f9a5804Test
https://doi.org/10.1016/j.jacbts.2019.10.003Test

المؤلفون: Schön, Christian, Becirovic, Elvir, Biel, Martin, Michalakis, Stylianos

المساهمون: Boon, Camiel J F, Wijnholds, Jan, Boon, C J F ( Camiel J F ), Wijnholds, J ( Jan )

المصدر: Schön, Christian; Becirovic, Elvir; Biel, Martin; Michalakis, Stylianos (2017). Design and Development of AAV-based Gene Supplementation Therapies for Achromatopsia and Retinitis Pigmentosa. In: Boon, Camiel J F; Wijnholds, Jan. Retinal Gene Therapy : Methods and Protocols. München: Springer, 33-46.

مصطلحات موضوعية: Ophthalmology Clinic, 610 Medicine & health, ACHM, Achromatopsia, Cone photoreceptors, Gene therapy, RP, Recombinant adeno-associated virus vectors, Retinitis pigmentosa, Rod photoreceptors, rAAV vectors

وصف الملف: application/pdf

العلاقة: https://www.zora.uzh.ch/id/eprint/254930/1/ZORA_254930.pdfTest; info:pmid/29188504; urn:isbn:978-1-4939-7521-1; urn:issn:1064-3745

الإتاحة: https://doi.org/10.5167/uzh-25493010.1007/978-1-4939-7522-8_3Test
https://www.zora.uzh.ch/id/eprint/254930Test/
https://www.zora.uzh.ch/id/eprint/254930/1/ZORA_254930.pdfTest