Gene therapy for spinal muscular atrophy: the Qatari experience.

Autor: 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.

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