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AAV-SPL 2.0, a Modified Adeno-Associated Virus Gene Therapy Agent for the Treatment of Sphingosine Phosphate Lyase Insufficiency Syndrome.
Khan, Ranjha; Oskouian, Babak; Lee, Joanna Y; Hodgin, Jeffrey B; Yang, Yingbao; Tassew, Gizachew; Saba, Julie D.
Affiliation
  • Khan R; Department of Pediatrics, Division of Hematology/Oncology, University of California, San Francisco, CA 94143, USA.
  • Oskouian B; Department of Pediatrics, Division of Hematology/Oncology, University of California, San Francisco, CA 94143, USA.
  • Lee JY; Department of Pediatrics, Division of Hematology/Oncology, University of California, San Francisco, CA 94143, USA.
  • Hodgin JB; Department of Pathology, University of Michigan School of Medicine, Ann Arbor, MI 48109, USA.
  • Yang Y; Department of Pathology, University of Michigan School of Medicine, Ann Arbor, MI 48109, USA.
  • Tassew G; Department of Pediatrics, Division of Hematology/Oncology, University of California, San Francisco, CA 94143, USA.
  • Saba JD; Department of Pediatrics, Division of Hematology/Oncology, University of California, San Francisco, CA 94143, USA.
Int J Mol Sci ; 24(21)2023 Oct 25.
Article in En | MEDLINE | ID: mdl-37958544
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Sphingosine / Genetic Therapy / Parvovirinae Limits: Animals / Humans Language: En Journal: Int J Mol Sci Year: 2023 Document type: Article Affiliation country: Country of publication:
Fulltext
Add to My VHL
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Sphingosine / Genetic Therapy / Parvovirinae Limits: Animals / Humans Language: En Journal: Int J Mol Sci Year: 2023 Document type: Article Affiliation country: Country of publication: