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.

MLPA analysis for molecular diagnosis of spinal muscular atrophy and correlation of 5q13.2 genes with disease phenotype in Egyptian patients.

Autor: Hassan, Heba A.¹ (AUTHOR) heba.amin@yahoo.com, Fahmy, Nagia A.² (AUTHOR), El-Bagoury, Nagham M.¹ (AUTHOR), Eissa, Noura R.¹ (AUTHOR), Sharaf-Eldin, Wessam E.¹ (AUTHOR), Issa, Mahmoud Y.³ (AUTHOR), Zaki, Maha S.³ (AUTHOR), Essawi, Mona L.¹ (AUTHOR)

Publikováno v: Egyptian Journal of Medical Human Genetics. 12/5/2022, Vol. 23 Issue 1, p1-9. 9p.

Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec -routine care evidence.

Autor: Aharoni S; Institute of Pediatric Neurology, Schneider Children's Medical Center of Israel, Petach Tikva, Israel. sharonah@clalit.org.il.; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel. sharonah@clalit.org.il., Bistritzer J; Institute of Pediatric Neurology, Schneider Children's Medical Center of Israel, Petach Tikva, Israel.; Pediatric Neurology Unit, Soroka University Medical Center, Ben Gurion University, Beer Sheva, Israel., Levine H; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.; Pulmonary Institute, Schneider Children's Medical Center, Petach Tikva, Israel., Sagi L; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.; Pediatric Neurology Institute, Dana-Dwek Children's Hospital, Tel Aviv Medical Center, Tel Aviv, Israel., Fattal-Valevski A; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.; Pediatric Neurology Institute, Dana-Dwek Children's Hospital, Tel Aviv Medical Center, Tel Aviv, Israel., Ginzberg M; Pediatric Neurology Unit, Wolfson Medical Center, Holon, Israel., Noyman I; Pediatric Neurology Unit, Soroka University Medical Center, Ben Gurion University, Beer Sheva, Israel., Cohen R; Institute of Pediatric Neurology, Schneider Children's Medical Center of Israel, Petach Tikva, Israel.; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel., Nevo Y; Institute of Pediatric Neurology, Schneider Children's Medical Center of Israel, Petach Tikva, Israel.; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

Publikováno v: Gene therapy [Gene Ther] 2023 Feb; Vol. 30 (1-2), pp. 101-106. Date of Electronic Publication: 2022 Apr 27.