Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches (Q59340096)

2

0 references

Nicolina Cristina Sorrentino

3

0 references

Bernhard Gentner

4

0 references

Alessandro Aiuti

5

0 references

Maria Ester Bernardo

Italian Journal of Paediatrics

6

0 references

language of work or name

English

0 references

publication date

16 November 2018

10 references

12 March 2019

14 March 2019

21 March 2019

24 March 2019

22 April 2019

25 April 2019

17 June 2019

25 June 2019

19 September 2019

29 October 2020

10 references

12 March 2019

14 March 2019

21 March 2019

24 March 2019

22 April 2019

25 April 2019

17 June 2019

25 June 2019

19 September 2019

29 October 2020

44

0 references

page(s)

130

0 references

issue

Suppl 2

0 references

copyright license

Creative Commons Attribution 4.0 International

10 references

12 March 2019

14 March 2019

21 March 2019

24 March 2019

22 April 2019

25 April 2019

17 June 2019

25 June 2019

19 September 2019

on focus list of Wikimedia project

29 October 2020

ScienceSource

0 references

exact match

https://scigraph.springernature.com/pub.10.1186/s13052-018-0565-y

0 references

cites work

Inborn errors of mucopolysaccharide metabolism

1 reference

12 December 2020

Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation

1 reference

12 December 2020

Drug and gene delivery to the brain: the vascular route

1 reference

12 December 2020

Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow

1 reference

12 December 2020

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

1 reference

12 December 2020

Cord-blood transplants from unrelated donors in patients with Hurler's syndrome

1 reference

12 December 2020

Prevention of neuropathology in the mouse model of Hurler syndrome

1 reference

12 December 2020

Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.

1 reference

12 December 2020

Enzyme-replacement therapy for metabolic storage disorders

1 reference

12 December 2020

Molecular biology of the blood-brain barrier

1 reference

12 December 2020

Gene therapy for lysosomal storage diseases.

1 reference

12 December 2020

Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII.

1 reference

12 December 2020

Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice

1 reference

12 December 2020

Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS.

1 reference

12 December 2020

Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes

1 reference

12 December 2020

The mucopolysaccharidoses: a success of molecular medicine.

1 reference

12 December 2020

The clinical outcome of Hurler syndrome after stem cell transplantation.

1 reference

12 December 2020

Umbilical cord blood from an unrelated donor as source for stem cell transplantations in inborn errors of metabolism

1 reference

12 December 2020

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

1 reference

12 December 2020

Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome

1 reference

12 December 2020

Restoration of central nervous system alpha-N-acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno-associated viral type 2 vector delivery

1 reference

12 December 2020

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.

1 reference

12 December 2020

Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy

1 reference

12 December 2020

Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer

1 reference

12 December 2020

Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.

1 reference

12 December 2020

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

1 reference

12 December 2020

Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system

1 reference

12 December 2020

Therapy for the mucopolysaccharidoses

1 reference

12 December 2020

Hurler Disease Bone Marrow Stromal Cells Exhibit Altered Ability to Support Osteoclast Formation

1 reference

12 December 2020

Enzyme replacement therapy for lysosomal diseases: lessons from 20 years of experience and remaining challenges

1 reference

12 December 2020

A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA.

1 reference

12 December 2020

Progress in gene therapy for neurological disorders.

1 reference

12 December 2020

Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease

1 reference

12 December 2020

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

1 reference

12 December 2020

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

1 reference

12 December 2020

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

1 reference

12 December 2020

Early initiation of enzyme replacement therapy for the mucopolysaccharidoses

1 reference

12 December 2020

Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial

1 reference

12 December 2020

Current and potential therapeutic strategies for mucopolysaccharidoses

1 reference

12 December 2020

Elosulfase alfa: first global approval

1 reference

12 December 2020

Insulin receptor antibody-sulfamidase fusion protein penetrates the primate blood-brain barrier and reduces glycosoaminoglycans in Sanfilippo type A cells

1 reference

12 December 2020

Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I

1 reference

12 December 2020

Lysosomal storage disorder screening implementation: findings from the first six months of full population pilot testing in Missouri

1 reference

12 December 2020

First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient

1 reference

12 December 2020

Therapies for the bone in mucopolysaccharidoses

1 reference

12 December 2020

Lysosomal storage diseases: from pathophysiology to therapy.

1 reference

12 December 2020

Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study.

1 reference

12 December 2020

Targeted delivery of protein and gene medicines through the blood-brain barrier

1 reference

12 December 2020

Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines.

1 reference

12 December 2020

Hematopoietic Stem Cell Gene Therapy Corrects Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II.

1 reference

12 December 2020

Clinical applications of gene therapy for primary immunodeficiencies

1 reference

12 December 2020

TREC Based Newborn Screening for Severe Combined Immunodeficiency Disease: A Systematic Review

1 reference

12 December 2020

Expanded newborn screening by mass spectrometry: New tests, future perspectives.

1 reference

12 December 2020

Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?

1 reference

12 December 2020

A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-associated Virus Serotypes Upon Cerebrospinal Fluid Administration in Pigs

1 reference

12 December 2020

A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome

1 reference

12 December 2020

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.

1 reference

12 December 2020

Pilot study of newborn screening for six lysosomal storage diseases using Tandem Mass Spectrometry

1 reference

12 December 2020

Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin.

1 reference

12 December 2020

Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.

1 reference

12 December 2020

Preclinical testing of the safety and tolerability of LV-mediated above normal alpha-L-iduronidase expression in murine and human hematopoietic cells using toxicology and biodistribution GLP studies

1 reference

12 December 2020

Brain Targeting in MPS-IIIA.

1 reference

12 December 2020

The role of conditioning in hematopoietic stem cell gene therapy.

1 reference

12 December 2020

Lentiviral vectors, two decades later

1 reference

12 December 2020

Gene therapy for metachromatic leukodystrophy.

1 reference

12 December 2020

Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy

1 reference

12 December 2020