المؤلفون: Ali HG; Department of Pharmacy, Hamad Medical Corporation, Doha, Qatar., Ibrahim K; Department of Pediatrics, Sidra Medicine, Doha, Qatar.; Weill Cornell Medical College, Doha, Qatar., Elsaid MF; Department of Pediatrics, Sidra Medicine, Doha, Qatar.; Weill Cornell Medical College, Doha, Qatar., Mohamed RB; Qatar Rehabilitation Institute, Hamad Medical Corporation, Doha, Qatar.; Department of Pediatrics, Hamad Medical Corporation, Doha, Qatar., Abeidah MIA; Qatar Rehabilitation Institute, Hamad Medical Corporation, Doha, Qatar.; Department of Pediatrics, Hamad Medical Corporation, Doha, Qatar., Al Rawwas AO; Qatar Rehabilitation Institute, Hamad Medical Corporation, Doha, Qatar.; Department of Pediatrics, Hamad Medical Corporation, Doha, Qatar., Elshafey K; Department of Pediatrics, Hamad Medical Corporation, Doha, Qatar., Almulla H; Department of Medical Genetics, Hamad Medical Corporation, Doha, Qatar., El-Akouri K; Department of Pediatrics, Sidra Medicine, Doha, Qatar.; Department of Medical Genetics, Hamad Medical Corporation, Doha, Qatar., Almulla M; Department of Pediatrics, Sidra Medicine, Doha, Qatar.; Department of Medical Genetics, Hamad Medical Corporation, Doha, Qatar., Othman A; Department of Medical Genetics, Hamad Medical Corporation, Doha, Qatar., Musa S; Department of Medical Genetics, Hamad Medical Corporation, Doha, Qatar., Al-Mesaifri F; Department of Pediatrics, Sidra Medicine, Doha, Qatar.; Department of Medical Genetics, Hamad Medical Corporation, Doha, Qatar., Ali R; Department of Pediatrics, Sidra Medicine, Doha, Qatar.; Department of Medical Genetics, Hamad Medical Corporation, Doha, Qatar.; Center of Rare Disease, Hamad Medical Corporation, Doha, Qatar., Shahbeck N; Department of Pediatrics, Sidra Medicine, Doha, Qatar.; Department of Medical Genetics, Hamad Medical Corporation, Doha, Qatar., Al-Mureikhi M; Department of Pediatrics, Sidra Medicine, Doha, Qatar.; Department of Medical Genetics, Hamad Medical Corporation, Doha, Qatar., Alsulaiman R; Department of Medical Genetics, Hamad Medical Corporation, Doha, Qatar.; Center of Rare Disease, Hamad Medical Corporation, Doha, Qatar., Alkaabi S; Center of Rare Disease, Hamad Medical Corporation, Doha, Qatar., Ben-Omran T; Department of Pediatrics, Sidra Medicine, Doha, Qatar. tawben11@hotmail.com.; Weill Cornell Medical College, Doha, Qatar. tawben11@hotmail.com.; Department of Medical Genetics, Hamad Medical Corporation, Doha, Qatar. tawben11@hotmail.com.; Center of Rare Disease, Hamad Medical Corporation, Doha, Qatar. tawben11@hotmail.com.

المصدر: Gene therapy [Gene Ther] 2021 Nov; Vol. 28 (10-11), pp. 676-680. Date of Electronic Publication: 2021 Jul 19.

نوع المنشور: Journal Article; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE

مواضيع طبية MeSH: Muscular Atrophy, Spinal*/drug therapy , Muscular Atrophy, Spinal*/therapy , Spinal Muscular Atrophies of Childhood*/drug therapy , Spinal Muscular Atrophies of Childhood*/therapy, Bilirubin ; Child ; Genetic Therapy ; Humans ; Infant ; Mutation