Cargando…

Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches

Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders caused by a deficiency in lysosomal enzymes catalyzing the stepwise degradation of glycosaminoglycans (GAGs). The current therapeutic strategies of enzyme replacement therapy and allogeneic hematopoietic stem cell transplantation...

Descripción completa

Detalles Bibliográficos
Autores principales:	Fraldi, Alessandro, Serafini, Marta, Sorrentino, Nicolina Cristina, Gentner, Bernhard, Aiuti, Alessandro, Bernardo, Maria Ester
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2018
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6238250/ https://www.ncbi.nlm.nih.gov/pubmed/30442177 http://dx.doi.org/10.1186/s13052-018-0565-y

Ejemplares similares

Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy
por: Zonari, Erika, et al.
Publicado: (2017)

A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA
por: Sorrentino, Nicolina Cristina, et al.
Publicado: (2013)

The EHA Research Roadmap: Hematopoietic Stem Cell Gene Therapy
por: Naldini, Luigi, et al.
Publicado: (2022)

Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series
por: Canarutto, Daniele, et al.
Publicado: (2021)

Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products
por: Aiuti, Alessandro, et al.
Publicado: (2017)

P1392: INTERIM ANALYSIS OF FIRST IN HUMAN PHASE I-II CLINICAL TRIAL OF EX-VIVO GENE THERAPY FOR HURLER SYNDROME: AN UPDATE AT 3 YEAR FOLLOW-UP
por: Tucci, Francesca, et al.
Publicado: (2023)

Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?
por: Tomatsu, Shunji, et al.
Publicado: (2015)

In vivo Gene Therapy to the Liver and Nervous System: Promises and Challenges
por: Cantore, Alessio, et al.
Publicado: (2022)

Mucopolysaccharidoses type I gene therapy
por: Hurt, Sarah C., et al.
Publicado: (2021)

Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art
por: de Castro, María José, et al.
Publicado: (2021)

In Vivo and Ex Vivo Gene Electrotransfer in Ophthalmological Disorders
por: Fusco, Roberta, et al.
Publicado: (2022)

An ex vivo gene therapy approach in X-linked retinoschisis
por: Bashar, Abu E., et al.
Publicado: (2016)

Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I
por: Penati, Rachele, et al.
Publicado: (2017)

Neonatal umbilical cord blood transplantation halts skeletal disease progression in the murine model of MPS-I
por: Azario, Isabella, et al.
Publicado: (2017)

Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease
por: Squeri, Giorgia, et al.
Publicado: (2019)

The Inflammation in the Cytopathology of Patients With Mucopolysaccharidoses- Immunomodulatory Drugs as an Approach to Therapy
por: Wiesinger, Anna-Maria, et al.
Publicado: (2022)

Assessing the Impact of Cyclosporin A on Lentiviral Transduction and Preservation of Human Hematopoietic Stem Cells in Clinically Relevant Ex Vivo Gene Therapy Settings
por: Petrillo, Carolina, et al.
Publicado: (2019)

Retinal Degeneration in MPS-IIIA Mouse Model
por: Intartaglia, Daniela, et al.
Publicado: (2020)

Development and clinical translation of ex vivo gene therapy
por: Wu, Xiaomo, et al.
Publicado: (2022)

ENT and mucopolysaccharidoses
por: Bianchi, Pier Marco, et al.
Publicado: (2018)

Diagnosis of Mucopolysaccharidoses
por: Kubaski, Francyne, et al.
Publicado: (2020)

Glaucoma in mucopolysaccharidoses
por: Kong, Weijing, et al.
Publicado: (2021)

Retroviral Integrations in Gene Therapy Trials
por: Biasco, Luca, et al.
Publicado: (2012)

Anesthesiological risks in mucopolysaccharidoses
por: Moretto, Alessandra, et al.
Publicado: (2018)

In Vivo Confocal Microscopy and Anterior Segment Optical Coherence Tomography Findings in Two Cases with Mucopolysaccharidoses
por: Karaküçük, Yalçın, et al.
Publicado: (2020)

Newborn screening in mucopolysaccharidoses
por: Donati, Maria Alice, et al.
Publicado: (2018)

Development of alternative gene transfer techniques for ex vivo and in vivo gene therapy in a canine model
por: Noda, Masashi, et al.
Publicado: (2021)

Antihyperglycemic Effect of Lavandula pedunculata: In Vivo, In Vitro and Ex Vivo Approaches
por: Boutahiri, Salima, et al.
Publicado: (2021)

Innovations Needed for Effective Implementation of Ex Vivo Gene Therapies
por: Cavazzana, Marina
Publicado: (2017)

Molecular purging of multiple myeloma cells by ex-vivo culture and retroviral transduction of mobilized-blood CD34(+ )cells
por: Deola, Sara, et al.
Publicado: (2007)

Pentosan Polysulfate: A Novel Therapy for the Mucopolysaccharidoses
por: Schuchman, Edward H., et al.
Publicado: (2013)

New treatments for the mucopolysaccharidoses: from pathophysiology to therapy
por: Fecarotta, Simona, et al.
Publicado: (2018)

Cathepsins in the Pathophysiology of Mucopolysaccharidoses: New Perspectives for Therapy
por: De Pasquale, Valeria, et al.
Publicado: (2020)

Novel Enzyme Replacement Therapies for Neuropathic Mucopolysaccharidoses
por: Sato, Yuji, et al.
Publicado: (2020)

Difficulties Associated with Enzyme Replacement Therapy for Mucopolysaccharidoses
por: Yıldız, Yılmaz, et al.
Publicado: (2021)

The new frame for Mucopolysaccharidoses
por: Parini, Rossella, et al.
Publicado: (2018)

Orthopaedic challenges for mucopolysaccharidoses
por: Borgo, Andrea, et al.
Publicado: (2018)

Pathogenesis of Mucopolysaccharidoses, an Update
por: Fecarotta, Simona, et al.
Publicado: (2020)

Ophthalmological Findings in Mucopolysaccharidoses
por: Tomatsu, Shizuka, et al.
Publicado: (2019)

Genome Editing for Mucopolysaccharidoses
por: Poletto, Edina, et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_f0ba8stf3i9b74vk7bs463sjde