نتائج البحث

SMA Treatment Innovations.

• Authors : Canpolat, Mehmet¹; Güleç, Ayten¹; Öztürk, Selcan¹

• Source: Gazi Medical Journal. 2024 Supplement, Vol. 35, p13-13. 1/2p.

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NEW THERAPIES IN SPINAL MUSCULAR ATROPHY.

• Authors : MIREA, A.^1,2; SHELBY, S.²; LEANCA, M. C.²

• Source: Balneo Research Journal. Sep2020, Vol. 11 Issue 3, pL66-L66. 1p.

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Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors

• Authors : Makoto Horiuchi; Christian J. Hinderer; Jenny A. Greig

Subjects: AAV; intrathecal; intravenous immunoglobulin

• Source: Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 272-280 (2022)

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Gene Therapy for ALS—A Perspective.

• Authors : Cappella, Marisa¹ (AUTHOR); Ciotti, Chiara¹ (AUTHOR); Cohen-Tannoudji, Mathilde¹ (AUTHOR)

Subjects: *GENE therapy; *AMYOTROPHIC lateral sclerosis; *SPINAL muscular atrophy

• Source: International Journal of Molecular Sciences. Sep2019, Vol. 20 Issue 18, p4388-4388. 1p. 2 Diagrams, 1 Chart.

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Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord

• Authors : Jensen, Thomas Leth; Gøtzsche, Casper René; Woldbye, David P.D.

Subjects: central nervous system; clinical trials; gene therapy

• Source: Jensen , T L , Gøtzsche , C R & Woldbye , D P D 2021 , ' Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord ' , Frontiers in Molecular

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