Cargando…

Gene Therapy Developments for Pompe Disease

Pompe disease is an inherited neuromuscular disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). The most severe form is infantile-onset Pompe disease, presenting shortly after birth with symptoms of cardiomyopathy, respiratory failure and skeletal muscle weakness. Lat...

Descripción completa

Detalles Bibliográficos
Autores principales:	Unnisa, Zeenath, Yoon, John K., Schindler, Jeffrey W., Mason, Chris, van Til, Niek P.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2022
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8869611/ https://www.ncbi.nlm.nih.gov/pubmed/35203513 http://dx.doi.org/10.3390/biomedicines10020302

Ejemplares similares

Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease
por: Dogan, Yildirim, et al.
Publicado: (2022)

Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease
por: Stok, Merel, et al.
Publicado: (2020)

High-throughput analysis of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing
por: Plasschaert, Robert N., et al.
Publicado: (2022)

Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease
por: Liang, Qiushi, et al.
Publicado: (2022)

IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy
por: Liang, Qiushi, et al.
Publicado: (2022)

Advancements in AAV-mediated Gene Therapy for Pompe Disease
por: Salabarria, S.M., et al.
Publicado: (2020)

Gene and Cellular Therapies for Leukodystrophies
por: Aerts-Kaya, Fatima, et al.
Publicado: (2023)

Pharmacological Chaperone Therapy for Pompe Disease
por: Borie-Guichot, Marc, et al.
Publicado: (2021)

Deficiency in Aryl Hydrocarbon Receptor (AHR) Expression throughout Aging Alters Gene Expression Profiles in Murine Long-Term Hematopoietic Stem Cells
por: Bennett, John A., et al.
Publicado: (2015)

Pompe Disease: New Developments in an Old Lysosomal Storage Disorder
por: Meena, Naresh K., et al.
Publicado: (2020)

The impact of Pompe disease on smooth muscle: a review
por: MCCALL, Angela L., et al.
Publicado: (2019)

Gene Augmentation and Editing to Improve TCR Engineered T Cell Therapy against Solid Tumors
por: Lo Presti, Vania, et al.
Publicado: (2020)

Newborn Screening for Pompe Disease
por: Sawada, Takaaki, et al.
Publicado: (2020)

Targeted approaches to induce immune tolerance for Pompe disease therapy
por: Doerfler, Phillip A, et al.
Publicado: (2016)

Development of Newborn Screening for Pompe Disease
por: Hwu, Wuh-Liang, et al.
Publicado: (2020)

The Pompe Registry: tracking Pompe disease symptoms in a broad patient population
por: Byrne, B, et al.
Publicado: (2008)

Enzyme replacement therapy and fatigue in adults with Pompe disease
por: Güngör, Deniz, et al.
Publicado: (2013)

Nanotechnology-based gene therapy as a credible tool in the treatment of Alzheimer’s disease
por: Unnisa, Aziz, et al.
Publicado: (2023)

Development of a disease severity scoring system for patients with Pompe disease
por: Giannini, E, et al.
Publicado: (2008)

The Respiratory Phenotype of Pompe Disease Mouse Models
por: Fusco, Anna F., et al.
Publicado: (2020)

Enzyme replacement therapy (ERT) in pompe disease
por: Fiumara, Agata
Publicado: (2014)

Hearing in adults with Pompe disease
por: van der Beek, Nadine A. M. E., et al.
Publicado: (2011)

Advances in Diagnosis and Management of Pompe Disease
por: Davison, James E.
Publicado: (2020)

Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease
por: Han, Sang-oh, et al.
Publicado: (2019)

Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease
por: Hordeaux, J., et al.
Publicado: (2017)

Teaching tolerance: New approaches to enzyme replacement therapy for Pompe disease
por: Cousens, Leslie P., et al.
Publicado: (2012)

A review of treatment of Pompe disease in infants
por: Chien, Yin-Hsiu, et al.
Publicado: (2007)

Pompe disease: pathogenesis, molecular genetics and diagnosis
por: Taverna, Simona, et al.
Publicado: (2020)

Pregnancy in Pompe disease
por: Plöckinger, Ursula
Publicado: (2013)

Familial Pompe Disease
por: Tecellioglu, Mehmet, et al.
Publicado: (2015)

Fatigue in neuromuscular disorders: focus on Guillain–Barré syndrome and Pompe disease
por: de Vries, J. M., et al.
Publicado: (2009)

Characteristics of Pompe disease in China: a report from the Pompe registry
por: Zhao, Yuying, et al.
Publicado: (2019)

Impact of enzyme replacement therapy on survival in adults with Pompe disease
por: Güngör, Deniz, et al.
Publicado: (2013)

Pompe disease: from pathophysiology to therapy and back again
por: Lim, Jeong-A, et al.
Publicado: (2014)

Limitations of muscle biopsy in Pompe disease
por: Vissing, John
Publicado: (2013)

A conceptual disease model for adult Pompe disease
por: Kanters, Tim A., et al.
Publicado: (2015)

Establishing Pompe Disease Newborn Screening: The Role of Industry
por: Keutzer, Joan M.
Publicado: (2020)

The Clinical Management of Pompe Disease: A Pediatric Perspective
por: Marques, Jorge Sales
Publicado: (2022)

A Comprehensive Update on Late-Onset Pompe Disease
por: Labella, Beatrice, et al.
Publicado: (2023)

Fatigue: an important feature of late-onset Pompe disease
por: Hagemans, M. L. C., et al.
Publicado: (2007)

Cannot write session to /tmp/vufind_sessions/sess_0894k2g72fm2l7jjn27kctjlfp