Gene Therapy for Childhood Neurological Disease

  • Mustafa Sahin
    Communications should be addressed to: Dr. Sahin; Department of Neurology; Translational Neuroscience Center; Boston Children's Hospital; 300 Longwood Avenue CLS14073; Boston, Massachusetts 02115.
    Department of Neurology, Translational Neuroscience Center, Boston Children's Hospital, Boston, Massachusetts
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      We are going through a transformational change in child neurology. The field is moving from description of symptoms and syndromes to design of rational therapy based on identified etiology of childhood neurological diseases. Advances in genetics and genomics have enabled us to find underlying genetic defects at a dizzying pace and greatly expanded the list of potential genes responsible for neuromuscular and neurodevelopmental diseases. The challenge now is to use this knowledge to design safe and effective treatment modalities for our patients.
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      Linked Article

      • Experimental Therapies in the Murine Model of Globoid Cell Leukodystrophy
        Pediatric NeurologyVol. 51Issue 5
        • Preview
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        • PDF
      • Gene Therapy for Muscular Dystrophy: Moving the Field Forward
        Pediatric NeurologyVol. 51Issue 5
        • Preview
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        • Full-Text
        • PDF