Cargando…

Hematopoietic Stem cell transplantation and lentiviral vector‐based gene therapy for Krabbe's disease: Present convictions and future prospects

Currently, presymtomatic hematopoietic stem and progenitor cell transplantation (HSPCT) is the only therapeutic modality that alleviates Krabbe's disease (KD)‐induced central nervous system damage. However, all HSPCT‐treated patients exhibit severe deterioration in peripheral nervous system fun...

Descripción completa

Detalles Bibliográficos
Autores principales:	Hu, Peirong, Li, Yedda, Nikolaishvili‐Feinberg, Nana, Scesa, Giuseppe, Bi, Yanmin, Pan, Dao, Moore, Dominic, Bongarzone, Ernesto R., Sands, Mark S., Miller, Ryan, Kafri, Tal
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2016
Materias:	Therapies and Clinical Management
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5027985/ https://www.ncbi.nlm.nih.gov/pubmed/27638600 http://dx.doi.org/10.1002/jnr.23847

Ejemplares similares

Generation of a stable packaging cell line producing high-titer PPT-deleted integration-deficient lentiviral vectors
por: Hu, Peirong, et al.
Publicado: (2015)

Krabbe disease: New hope for an old disease
por: Bradbury, Allison M., et al.
Publicado: (2021)

New therapeutic approaches for Krabbe disease: The potential of pharmacological chaperones
por: Spratley, Samantha J., et al.
Publicado: (2016)

Psychosine enhances the shedding of membrane microvesicles: Implications in demyelination in Krabbe’s disease
por: D’Auria, Ludovic, et al.
Publicado: (2017)

Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization.
por: Hu, Peirong, et al.
Publicado: (2018)

The Pathogenic Sphingolipid Psychosine is Secreted in Extracellular Vesicles in the Brain of a Mouse Model of Krabbe Disease
por: Reiter, Cory R., et al.
Publicado: (2022)

CRISPR-Cas9 Knock-In of T513M and G41S Mutations in the Murine β–Galactosyl-Ceramidase Gene Re-capitulates Early-Onset and Adult-Onset Forms of Krabbe Disease
por: Rebiai, Rima, et al.
Publicado: (2022)

Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells
por: Zhang, Fang, et al.
Publicado: (2007)

Brainstem development requires galactosylceramidase and is critical for pathogenesis in a model of Krabbe disease
por: Weinstock, Nadav I., et al.
Publicado: (2020)

Communicate with mastery: speak with conviction and write for impact
por: Schramm, J D, et al.
Publicado: (2020)

Toward Personalized Gene Therapy: Characterizing the Host Genetic Control of Lentiviral-Vector-Mediated Hepatic Gene Delivery
por: Suwanmanee, Thipparat, et al.
Publicado: (2017)

Benefits of newborn screening and hematopoietic cell transplant in infantile Krabbe disease
por: Page, Kristin M., et al.
Publicado: (2022)

Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease
por: Heller, Gregory J., et al.
Publicado: (2021)

Cultivez votre charisme: et développez votre pouvoir de conviction
por: Hills, Chilina
Publicado: (2005)

The leader's guide to speaking with presence: how to project confidence, conviction, and authority
por: Baldoni, John
Publicado: (2014)

Problem solved: a powerful system for making complex decisions with confidence and conviction
por: Strauss Einhorn, Cheryl, et al.
Publicado: (2017)

CD14(+)/CD31(+) monocytes expanded by UM171 correct hemophilia A in zebrafish upon lentiviral gene transfer of factor VIII
por: Elnaggar, Muhammad, et al.
Publicado: (2022)

Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe‐affected non‐human primates by intracerebral lentiviral gene therapy
por: Meneghini, Vasco, et al.
Publicado: (2016)

Convict Marriages
por: Barker, F. A.
Publicado: (1912)

Deceased Convicts
Publicado: (1856)

Neuroimmune mechanisms in Krabbe's disease
por: Potter, Gregory B., et al.
Publicado: (2016)

La conviction idéologique /
por: Moreux, Colette
Publicado: (1978)

Children convicted of homicide.
por: Walsh-Brennan, K. S.
Publicado: (1975)

Convict Marriages in the Andamans
por: Woolley, J. M.
Publicado: (1912)

Diseases of Overfed Convicts
Publicado: (1856)

Another Conviction for Substitution
Publicado: (1905)

Medical Service for Convicts
Publicado: (1886)

“Alba” Dentists Convicted
Publicado: (1903)

Advances in the Diagnosis and Treatment of Krabbe Disease
por: Wenger, David A, et al.
Publicado: (2021)

Lipid nanoparticles allow efficient and harmless ex vivo gene editing of human hematopoietic cells
por: Vavassori, Valentina, et al.
Publicado: (2023)

237. Lentiviral Vector Gene Transfer to Porcine Sinus and Pulmonary Airways
Publicado: (2012)

The Relational Costs of Wrongful Convictions
por: Umamaheswar, Janani
Publicado: (2023)

Long-term follow-up of post hematopoietic stem cell transplantation for Hurler syndrome: Clinical, biochemical, and pathological improvements
por: Yasuda, Eriko, et al.
Publicado: (2015)

Azasugar inhibitors as pharmacological chaperones for Krabbe disease
por: Hill, Chris H., et al.
Publicado: (2015)

Gene therapy for CNS diseases – Krabbe disease
por: Rafi, Mohammad A.
Publicado: (2016)

Krabbe disease: A personal perspective and hypothesis
por: Rafi, Mohammad A.
Publicado: (2022)

Neurodegenerative Disorder Risk in Krabbe Disease Carriers
por: Vantaggiato, Lorenza, et al.
Publicado: (2022)

The Effects of Antipsychotics in Experimental Models of Krabbe Disease
por: Sharma, Kapil, et al.
Publicado: (2023)

Globoid Cell Leukodystrophy (Krabbe Disease): An Update
por: Maghazachi, Azzam A
Publicado: (2023)

Hypoxia-inducible lentiviral gene expression in engineered human macrophages
por: Chinn, Harrison K, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_387v8m95h8stisbm3sg5pp3kgj