Cargando…

Parkinson's Disease Gene Therapy: Success by Design Meets Failure by Efficacy

Over the past decade, nine gene therapy clinical trials for Parkinson's disease (PD) have been initiated and completed. Starting with considerable optimism at the initiation of each trial, none of the programs has yet borne sufficiently robust clinical efficacy or found a clear path toward regu...

Descripción completa

Detalles Bibliográficos
Autores principales:	Bartus, Raymond T, Weinberg, Marc S, Samulski, R. Jude
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2014
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3944322/ https://www.ncbi.nlm.nih.gov/pubmed/24356252 http://dx.doi.org/10.1038/mt.2013.281

Ejemplares similares

Gene therapy for Parkinson's disease: a decade of progress supported by posthumous contributions from volunteer subjects
por: Bartus, Raymond T.
Publicado: (2015)

Long-term post-mortem studies following neurturin gene therapy in patients with advanced Parkinson’s disease
por: Chu, Yaping, et al.
Publicado: (2020)

Gene therapy approaches for equine osteoarthritis
por: Thampi, Parvathy, et al.
Publicado: (2022)

Corrigendum: Gene therapy approaches for equine osteoarthritis
por: Thampi, Parvathy, et al.
Publicado: (2023)

Creating a Novel Origin of Replication through Modulating DNA-Protein Interfaces
por: Hewitt, F. Curtis, et al.
Publicado: (2010)

AAV Gene Therapy for MPS1-associated Corneal Blindness
por: Vance, Melisa, et al.
Publicado: (2016)

Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease
por: Francis, Jeremy S., et al.
Publicado: (2021)

Gene Therapy for Parkinson's Disease
por: Denyer, Rachel, et al.
Publicado: (2012)

Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial
por: Monahan, Paul E., et al.
Publicado: (2015)

Efficacy and safety of light therapy for Parkinson disease
por: Li, Yanghong, et al.
Publicado: (2022)

Adeno-Associated Virus (AAV) Versus Immune Response
por: Rabinowitz, Joseph, et al.
Publicado: (2019)

Global CNS Gene Delivery and Evasion of Anti-AAV Neutralizing Antibodies by Intrathecal AAV Administration in Non-Human Primates
por: Gray, Steven J., et al.
Publicado: (2013)

The efficacy of a “cocktail therapy” on Parkinson’s disease with dementia
por: Zhang, Chenhao, et al.
Publicado: (2019)

Gene Therapy for Parkinson’s Disease, An Update
por: Axelsen, Tobias M., et al.
Publicado: (2018)

Silencing of Parkinson's disease-associated genes with artificial mirtron mimics of miR-1224
por: Sibley, Christopher R., et al.
Publicado: (2012)

Oral Health Disorders in Parkinson’s Disease: More than Meets the Eye
por: Auffret, Manon, et al.
Publicado: (2021)

Prediction of Adeno-Associated Virus Neutralizing Antibody Activity for Clinical Application
por: Wang, Mei, et al.
Publicado: (2015)

Organizational Meeting Orientation: Setting the Stage for Team Success or Failure Over Time
por: Mroz, Joseph E., et al.
Publicado: (2019)

ROS networks: designs, aging, Parkinson’s disease and precision therapies
por: N. Kolodkin, Alexey, et al.
Publicado: (2020)

Opicapone Efficacy and Tolerability in Parkinson's Disease Patients Reporting Insufficient Benefit/Failure of Entacapone
por: Vokurka, Petr, et al.
Publicado: (2020)

The current status of gene therapy for Parkinson's disease
por: Muramatsu, Shin-ichi
Publicado: (2010)

Self-complementarity in adeno-associated virus enhances transduction and gene expression in mouse cochlear tissues
por: Casey, Graham, et al.
Publicado: (2020)

β-Cell–Specific IL-2 Therapy Increases Islet Foxp3(+)Treg and Suppresses Type 1 Diabetes in NOD Mice
por: Johnson, Mark C., et al.
Publicado: (2013)

Perspective on the Road toward Gene Therapy for Parkinson’s Disease
por: Blits, Bas, et al.
Publicado: (2017)

Current Experimental Studies of Gene Therapy in Parkinson's Disease
por: Lin, Jing-ya, et al.
Publicado: (2017)

A Review of Genetic and Gene Therapy for Parkinson’s Disease
por: Dumbhare, Omkar, et al.
Publicado: (2023)

Adeno-Associated Viral Vectors Show Serotype Specific Transduction of Equine Joint Tissue Explants and Cultured Monolayers
por: Hemphill, Daniel D., et al.
Publicado: (2014)

AAV Recombineering with Single Strand Oligonucleotides
por: Hirsch, Matthew L., et al.
Publicado: (2009)

AAV Exploits Subcellular Stress Associated with Inflammation, Endoplasmic Reticulum Expansion, and Misfolded Proteins in Models of Cystic Fibrosis
por: Johnson, Jarrod S., et al.
Publicado: (2011)

An update on advanced therapies for Parkinson's disease: From gene therapy to neuromodulation
por: Serva, Stephanie N., et al.
Publicado: (2022)

The Disease Modification Conundrum in Parkinson’s Disease: Failures and Hopes
por: Mari, Zoltan, et al.
Publicado: (2022)

Out FOXing Parkinson Disease: Where Development Meets Neurodegeneration
por: Wexler, Eric M, et al.
Publicado: (2007)

Stepping up to meet the challenge of freezing of gait in Parkinson’s disease
por: Lewis, Simon, et al.
Publicado: (2022)

Successful glucose lowering therapy triumphs in heart failure
por: Rossing, Peter
Publicado: (2021)

Therapy of Parkinson's Disease
por: Holzer, Jacob C.
Publicado: (1990)

The failure of success
por: Marrow, Alfred J
Publicado: (1972)

Planning successful meetings and events
por: Boehme, Ann J
Publicado: (1998)

Edging closer to successful gene therapy for Wilson disease
por: Sandahl, Thomas Damgaard, et al.
Publicado: (2022)

Sensorimotor rhythm neurofeedback as adjunct therapy for Parkinson's disease
por: Philippens, Ingrid H. C. H. M., et al.
Publicado: (2017)

Efficacy of cognitive rehabilitation in Parkinson's disease
por: Díez-Cirarda, María, et al.
Publicado: (2018)

Cannot write session to /tmp/vufind_sessions/sess_k3gm0e62nko3or3us0ioo22pcb