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Gene Therapy for Dopamine Dyshomeostasis: From Parkinson's to Primary Neurotransmitter Diseases.
Ng, Joanne; Barral, Serena; Waddington, Simon N; Kurian, Manju A.
Afiliación
  • Ng J; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London, United Kingdom.
  • Barral S; Genetic Therapy Accelerator Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, University College London, London, United Kingdom.
  • Waddington SN; Developmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, GOS-Institute of Child Health, University College London, London, United Kingdom.
  • Kurian MA; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London, United Kingdom.
Mov Disord ; 38(6): 924-936, 2023 06.
Article en En | MEDLINE | ID: mdl-37147851
ABSTRACT
Neurological disorders encompass a broad range of neurodegenerative and neurodevelopmental diseases that are complex and almost universally without disease modifying treatments. There is, therefore, significant unmet clinical need to develop novel therapeutic strategies for these patients. Viral gene therapies are a promising approach, where gene delivery is achieved through viral vectors such as adeno-associated virus and lentivirus. The clinical efficacy of such gene therapies has already been observed in two neurological disorders of pediatric onset; for spinal muscular atrophy and aromatic L-amino acid decarboxylase (AADC) deficiency, gene therapy has significantly modified the natural history of disease in these life-limiting neurological disorders. Here, we review recent advances in gene therapy, focused on the targeted delivery of dopaminergic genes for Parkinson's disease and the primary neurotransmitter disorders, AADC deficiency and dopamine transporter deficiency syndrome (DTDS). Although recent European Medicines Agency and Medicines and Healthcare products Regulatory Agency approval of Upstaza (eladocagene exuparvovec) signifies an important landmark, numerous challenges remain. Future research will need to focus on defining the optimal therapeutic window for clinical intervention, better understanding of the duration of therapeutic efficacy, and improved brain targeting. © 2023 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedad de Parkinson / Dopamina Límite: Child / Humans Idioma: En Revista: Mov Disord Asunto de la revista: NEUROLOGIA Año: 2023 Tipo del documento: Article País de afiliación: Reino Unido

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedad de Parkinson / Dopamina Límite: Child / Humans Idioma: En Revista: Mov Disord Asunto de la revista: NEUROLOGIA Año: 2023 Tipo del documento: Article País de afiliación: Reino Unido
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