Making Strides Towards Gene Therapy for Inherited Neurometabolic Diseases / Fare passi avanti verso la terapia genica per le malattie neurometaboliche ereditarie
Making Strides Towards Gene Therapy for Inherited Neurometabolic Diseases / Fare passi avanti verso la terapia genica per le malattie neurometaboliche ereditarie
Segnalato dal Dott. Giuseppe Cotellessa / Reported by Dr. Giuseppe Cotellessa
Researchers are making great strides toward developing gene-based strategies to treat a variety of inherited neurometabolic diseases characterized by severe neurological involvement. A review of the approaches currently under preclinical or clinical investigation is published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.
In the article entitled "Gene-Based Approaches to Inherited Neurometabolic Diseases," coauthors Valentina Poletti and Alessandra Biffi, Dana-Farber/Boston Children's Can-cer and Blood Disorders Center (Boston, MA), and University of Padova (Italy), describe the characteristics needed for a gene therapy to treat neurometabolic diseases to be successful.
The researchers focus in particular on progress made in the development of gene-based approaches to treat X-linked adrenoleukodystrophy (ALD), metachromatic leu-kodystropy (MLD), mucopolysaccharidoses (MPSs), and neuronal ceroid liposuscinoses (NCLs). They identify microglia as emerging key players in inherited neurodegenerative diseases, and therefore as important targets for achieving therapeutic efficacy in gene-based strategies.
"Drs. Poletti and Biffi document some of the dramatic clinical benefits that gene therapy has brought to infants and children with neurometabolic disorders," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA. "The breakthrough was the insight to use genetic alteration of bone marrow stem cells to cure diseases within the brain, using microglia as the vehicle. This platform has brought hope to many families with previously incurable diseases."
In the article entitled "Gene-Based Approaches to Inherited Neurometabolic Diseases," coauthors Valentina Poletti and Alessandra Biffi, Dana-Farber/Boston Children's Can-cer and Blood Disorders Center (Boston, MA), and University of Padova (Italy), describe the characteristics needed for a gene therapy to treat neurometabolic diseases to be successful.
The researchers focus in particular on progress made in the development of gene-based approaches to treat X-linked adrenoleukodystrophy (ALD), metachromatic leu-kodystropy (MLD), mucopolysaccharidoses (MPSs), and neuronal ceroid liposuscinoses (NCLs). They identify microglia as emerging key players in inherited neurodegenerative diseases, and therefore as important targets for achieving therapeutic efficacy in gene-based strategies.
"Drs. Poletti and Biffi document some of the dramatic clinical benefits that gene therapy has brought to infants and children with neurometabolic disorders," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA. "The breakthrough was the insight to use genetic alteration of bone marrow stem cells to cure diseases within the brain, using microglia as the vehicle. This platform has brought hope to many families with previously incurable diseases."
ITALIANO
I ricercatori stanno facendo grandi passi avanti nello sviluppo di strategie basate sui geni per trattare una varietà di malattie neurometaboliche ereditarie caratterizzate da un grave coinvolgimento neurologico. Una revisione degli approcci attualmente sotto indagine preclinica o clinica è pubblicata su Human Gene Therapy, una rivista peer-reviewed di Mary Ann Liebert, Inc., editori.
Nell'articolo intitolato "Approcci basati sui geni alle malattie neurometaboliche ereditarie", descrivono i coautori Valentina Poletti e Alessandra Biffi, del Centro per i disturbi del sangue e dei bambini Dana-Farber / Boston (Boston, MA) e l'Università di Padova (Italia). le caratteristiche necessarie per una terapia genica per il trattamento delle malattie neurometaboliche per avere successo.
I ricercatori si concentrano in particolare sui progressi compiuti nello sviluppo di approcci basati sul gene per il trattamento dell'adrenoleucodistrofia legata all'X (ALD), della leucododropia metacromatica (MLD), delle mucopolisaccaridosi (MPS) e delle liposuscinosi ceroidi neuronali (NCL). Identificano la microglia come attori chiave emergenti nelle malattie neurodegenerative ereditarie e quindi come obiettivi importanti per raggiungere l'efficacia terapeutica nelle strategie basate sui geni.
"I dott. Poletti e Biffi documentano alcuni dei drammatici benefici clinici che la terapia genica ha portato a neonati e bambini con disturbi neurometabolici", afferma il caporedattore Terence R. Flotte, MD, Celia e Isaac Haidak, professore di educazione medica e preside , Provost, e vice cancelliere esecutivo, Università del Massachusetts Medical School, Worcester, MA. "La svolta è stata l'intuizione di utilizzare l'alterazione genetica delle cellule staminali del midollo osseo per curare le malattie del cervello, usando la microglia come veicolo. Questa piattaforma ha portato speranza a molte famiglie con malattie precedentemente incurabili."
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