Novel Vector for Making Transgenic Animals for Genetic Regulation

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Robert Nicholls
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Gene Therapy for Prader-Willi Syndrome
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Developed using novel mini promoters to enhance gene expression and a PWS-minigene adeno-associated virus (AAV) vector, it may be possible to treat this neurodevelopmental disorder.
• Developmental disorders involving multiple genes.
Given the large number of genes involved in PWS, traditional gene therapy is not a feasible option due to limits in delivering more than one gene using AAV vectors.
Therapeutic Modality
Gene Therapy - AAV
Endocrinology and Metabolic Diseases
Prader-Willi Syndrome
Platform Technology
Robert Nicholls
Med-Pediatrics
2022-12-06
    • Technology Type
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        Therapeutic Modality
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    • Technology Subtype
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        Gene Therapy - AAV
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    • Therapeutic Areas
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        Endocrinology and Metabolic Diseases
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    • Therapeutic Indications
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        Prader-Willi Syndrome
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    • Tags
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        Platform Technology
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    • Lead Inventor
      • 3
        Kyle Orwig
      • 2
        Lawrence Kane
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        Alejandro Soto Gutierrez
      • 1
        Alexander Deiters
      • 1
        Brett Kaufman
      • 1
        Robert Nicholls
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    • Department
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        Med-Pediatrics
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    • All Tech Innovators
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        Robert David Nicholls
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