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AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies.
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AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies.
Yang H, Brown RH, Wang D, Strauss KA, Gao G. AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies. Trends Mol Med. 2021 06; 27(6):520-523.
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PubMed
subject areas
Dependovirus
Epilepsy
Genetic Therapy
Genetic Vectors
Hereditary Sensory and Autonomic Neuropathies
Humans
Phenotype
Recombinant Proteins
Sialyltransferases
Spastic Paraplegia, Hereditary
authors with profiles
Dan Wang PhD
Kevin Strauss MD
Robert H Brown MD, DPhil
Guangping Gao PhD