Advertisement

Gene Therapy for Childhood Neurological Disease

  • Mustafa Sahin
    Correspondence
    Communications should be addressed to: Dr. Sahin; Department of Neurology; Translational Neuroscience Center; Boston Children's Hospital; 300 Longwood Avenue CLS14073; Boston, Massachusetts 02115.
    Affiliations
    Department of Neurology, Translational Neuroscience Center, Boston Children's Hospital, Boston, Massachusetts
    Search for articles by this author
      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.
      To read this article in full you will need to make a payment

      Purchase one-time access:

      Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online access
      One-time access price info
      • For academic or personal research use, select 'Academic and Personal'
      • For corporate R&D use, select 'Corporate R&D Professionals'

      Subscribe:

      Subscribe to Pediatric Neurology
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect

      References

        • Gao G.
        • Vandenberghe L.H.
        • Wilson J.M.
        New recombinant serotypes of AAV vectors.
        Curr Gene Ther. 2005; 5: 285-297
        • Al-Zaidy S.
        • Rodino-Klapac L.
        • Mendell J.R.
        Gene therapy for muscular dystrophy: moving the field forward.
        Pediatr Neurol. 2014; 51: 607-618
        • Drachman D.B.
        • Toyka K.V.
        • Myer E.
        Prednisone in Duchenne muscular dystrophy.
        Lancet. 1974; 2: 1409-1412
        • Li Y.
        • Sands M.S.
        Experimental therapies in the murine model of globoid cell leukodystrophy.
        Pediatr Neurol. 2014; 51: 600-606
        • Sands M.S.
        • Davidson B.L.
        Gene therapy for lysosomal storage diseases.
        Mol Ther. 2006; 13: 839-849

      Linked Article

      • Experimental Therapies in the Murine Model of Globoid Cell Leukodystrophy
        Pediatric NeurologyVol. 51Issue 5
        • Preview
          Globoid cell leukodystrophy or Krabbe disease, is a rapidly progressive childhood lysosomal storage disorder caused by a deficiency in galactocerebrosidase. Galactocerebrosidase deficiency leads to the accumulation of galactosylsphingosine (psychosine), a cytotoxic lipid especially damaging to oligodendrocytes and Schwann cells. The progressive loss of cells involved in myelination results in a dysmyelinating phenotype affecting both the central and peripheral nervous systems. Current treatment for globoid cell leukodystrophy is limited to bone marrow or umbilical cord blood transplantation.
        • Full-Text
        • PDF
      • Gene Therapy for Muscular Dystrophy: Moving the Field Forward
        Pediatric NeurologyVol. 51Issue 5
        • Preview
          Gene therapy for the muscular dystrophies has evolved as a promising treatment for this progressive group of disorders. Although corticosteroids and/or supportive treatments remain the standard of care for Duchenne muscular dystrophy, loss of ambulation, respiratory failure, and compromised cardiac function is the inevitable outcome. Recent developments in genetically mediated therapies have allowed for personalized treatments that strategically target individual muscular dystrophy subtypes based on disease pathomechanism and phenotype.
        • Full-Text
        • PDF