Showing 1 - 8 results of 8 for search 'Deeksha Bali', query time: 0.03s Refine Results
  1. 1
    Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease

    Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease by Gensheng Wang, Sarah P Young, Deeksha Bali, Julie Hutt, Songtao Li, Janet Benson, Dwight D Koeberl

    Published 2014-01-01
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  2. 2
    Corrigendum to âAssessment of toxicity and biodistribution of recombinant AAV8 vectorâmediated immunomodulatory gene therapy in mice with Pompe diseaseâ

    Corrigendum to âAssessment of toxicity and biodistribution of recombinant AAV8 vectorâmediated immunomodulatory gene therapy in mice with Pompe diseaseâ by Gensheng Wang, Sarah P Young, Deeksha Bali, Julie Hutt, Songtao Li, Janet Benson, Dwight D Koeberl

    Published 2015-01-01
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  3. 3
    Screening data from 19 patients with late‐onset Pompe disease for a phase I clinical trial of AAV8 vector‐mediated gene therapy

    Screening data from 19 patients with late‐onset Pompe disease for a phase I clinical trial of AAV8 vector‐mediated gene therapy by William B. Hannah, Laura E. Case, Edward C. Smith, Crista Walters, Deeksha Bali, Priya S. Kishnani, Dwight D. Koeberl

    Published 2023-09-01
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  4. 4
    P173: Establishing preclinical efficacy for the first AAV gene therapy for GSD IX γ2 across stages of progressive liver disease

    P173: Establishing preclinical efficacy for the first AAV gene therapy for GSD IX γ2 across stages of progressive liver disease by Rebecca Gibson, Jeong-A Lim, Su Jin Choi, Neha Jumani, Deeksha Bali, Sarah Young, William Jeck, Baodong Sun, Aravind Asokan, Priya Kishnani

    Published 2023-01-01
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  5. 5
    P252: Development of a scoring system to define lysosomal diseases

    P252: Development of a scoring system to define lysosomal diseases by Matheus Wilke, Jenny Goldstein, Emily Groopman, Shruthi Mohan, Amber Waddell, Raquel Fernandez, Hongjie Chen, Deeksha Bali, Heather Baudet, Lorne Clarke, Christina Hung, Rong Mao, Tatiana Yuzyuk, William Craigen, Filippo Pinto e Vairo

    Published 2024-01-01
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  6. 6
    O39: The ClinGen Lysosomal Diseases Variant Curation Expert Panel’s guidance on classification of IDUA variants for mucopolysaccharidosis type I

    O39: The ClinGen Lysosomal Diseases Variant Curation Expert Panel’s guidance on classification of IDUA variants for mucopolysaccharidosis type I by Jenny Goldstein, Amber Waddell, Carlos Aschoff, Xiangwen Chen-Deutsch, Matthew Ellinwood, Roberto Mendez, Raquel Fernandez, Deeksha Bali, Troy Lund, Laura Pollard, Richard Steet, Filippo Pinto e Vairo, Timothy Wood, Lorne Clarke, Catherine Rehder

    Published 2024-01-01
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  7. 7
    P695: Uncertainty in interpretation of GAA variants detected through newborn screening without clinical manifestation of Pompe disease

    P695: Uncertainty in interpretation of GAA variants detected through newborn screening without clinical manifestation of Pompe disease by Dona Kanavy, Jenny Goldstein, Filippo Pinto e Vairo, Deeksha Bali, Xiangwen Chen-Deutsch, Taraka Donti, Shelly Goomber, Jennifer McGlaughon, Yinghong Pan, Bryce Seifert, Raquel Fernandez, Emily Kyle, Meredith Weaver, Lorne Clarke, Catherine Rehder

    Published 2024-01-01
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  8. 8
    O18: A path forward for patients with glycogen branching enzyme deficiency: Consensus on diagnosing and managing glycogen storage disease type IV*

    O18: A path forward for patients with glycogen branching enzyme deficiency: Consensus on diagnosing and managing glycogen storage disease type IV* by Rebecca Koch, Claudia Soler-Alfonso, Bridget Kiely, Akihiro Asai, Ariana Smith, Deeksha Bali, Peter Kang, Andrew Landstrom, H. Orhan Akman, T. Andrew Burrow, Jennifer Orthmann-Murphy, Deberah Goldman, Surekha Pendyal, Areeg El-Gharbawy, Stephanie Austin, Laura Case, Raphael Schiffmann, Michio Hirano, Priya Kishnani

    Published 2023-01-01
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