[HTML][HTML] Krabbe disease successfully treated via monotherapy of intrathecal gene therapy
AM Bradbury, JH Bagel, D Nguyen… - The Journal of …, 2020 - Am Soc Clin Investig
Globoid cell leukodystrophy (GLD; Krabbe disease) is a progressive, incurable neurodegenerative
disease caused by deficient activity of the hydrolytic enzyme galactosylceramidase (…
disease caused by deficient activity of the hydrolytic enzyme galactosylceramidase (…
Adeno-associated virus gene therapy in a sheep model of Tay–Sachs disease
…, BL Brunson, M Hwang, LE Ellis, AM Bradbury… - Human Gene …, 2018 - liebertpub.com
Tay–Sachs disease (TSD) is a fatal neurodegenerative disorder caused by a deficiency of
the enzyme hexosaminidase A (HexA). TSD also occurs in sheep, the only experimental …
the enzyme hexosaminidase A (HexA). TSD also occurs in sheep, the only experimental …
AAV-based in vivo gene therapy for neurological disorders
Q Ling, JA Herstine, A Bradbury, SJ Gray - Nature Reviews Drug …, 2023 - nature.com
Recent advancements in gene supplementation therapy are expanding the options for the
treatment of neurological disorders. Among the available delivery vehicles, adeno-associated …
treatment of neurological disorders. Among the available delivery vehicles, adeno-associated …
Direct intracranial injection of AAVrh8 encoding monkey β-N-acetylhexosaminidase causes neurotoxicity in the primate brain
GM2 gangliosidoses, including Tay–Sachs disease and Sandhoff disease, are lysosomal
storage disorders caused by deficiencies in β-N-acetylhexosaminidase (Hex). Patients are …
storage disorders caused by deficiencies in β-N-acetylhexosaminidase (Hex). Patients are …
[HTML][HTML] Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy
AM Bradbury, JN Cochran, VJ McCurdy, AK Johnson… - Molecular Therapy, 2013 - cell.com
Salutary responses to adeno-associated viral (AAV) gene therapy have been reported in the
mouse model of Sandhoff disease (SD), a neurodegenerative lysosomal storage disease …
mouse model of Sandhoff disease (SD), a neurodegenerative lysosomal storage disease …
[HTML][HTML] Krabbe disease: New hope for an old disease
AM Bradbury, ER Bongarzone, MS Sands - Neuroscience letters, 2021 - Elsevier
Krabbe disease (globoid cell leukodystrophy) is a lysosomal storage disease (LSD) characterized
by progressive and profound demyelination. Infantile, juvenile and adult-onset forms …
by progressive and profound demyelination. Infantile, juvenile and adult-onset forms …
AAVrh10 gene therapy ameliorates central and peripheral nervous system disease in canine globoid cell leukodystrophy (Krabbe disease)
AM Bradbury, MA Rafi, JH Bagel, BK Brisson… - Human Gene …, 2018 - liebertpub.com
Globoid cell leukodystrophy (GLD), or Krabbe disease, is an inherited, neurologic disorder
that results from deficiency of a lysosomal enzyme, galactosylceramidase. Most commonly, …
that results from deficiency of a lysosomal enzyme, galactosylceramidase. Most commonly, …
[HTML][HTML] CD8+ T cell depletion prevents neuropathology in a mouse model of globoid cell leukodystrophy
…, ER Jellison, AM Nicaise, AM Bradbury… - Journal of Experimental …, 2023 - rupress.org
Globoid cell leukodystrophy (GLD) or Krabbe’s disease is a fatal genetic demyelinating
disease of the central nervous system caused by loss-of-function mutations in the …
disease of the central nervous system caused by loss-of-function mutations in the …
AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system
…, AK Johnson, AN Randle, AM Bradbury… - ASN …, 2015 - journals.sagepub.com
Sandhoff disease (SD) is an autosomal recessive neurodegenerative disease caused by a
mutation in the gene for the β-subunit of β-N-acetylhexosaminidase (Hex), resulting in the …
mutation in the gene for the β-subunit of β-N-acetylhexosaminidase (Hex), resulting in the …
[HTML][HTML] Focus: Comparative Medicine: Canine and feline models of human genetic diseases and their contributions to advancing clinical therapies
BL Gurda, AM Bradbury, CH Vite - The Yale journal of biology and …, 2017 - ncbi.nlm.nih.gov
For many lethal or debilitating genetic disorders in patients there are no satisfactory therapies.
Several barriers exist that hinder the developments of effective therapies including the …
Several barriers exist that hinder the developments of effective therapies including the …