Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID

Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC

(2016) Sara Marcó et al.   Disease Models & Mechanisms

Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice

(2016) Christian Hinderer et al.   HUMAN GENE THERAPY

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

(2016) Maria Sessa et al.   LANCET

Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII

(2016) Brittney L Gurda et al.   MOLECULAR THERAPY

AAV9-mediated central nervous system–targeted gene delivery via cisterna magna route in mice

(2016) Vera Lukashchuk et al.   Molecular Therapy-Methods & Clinical Development

Adeno-associated virus serotypes for gene therapeutics

(2015) Leszek Lisowski et al.   CURRENT OPINION IN PHARMACOLOGY

A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II

(2015) Joseph Muenzer et al.   GENETICS IN MEDICINE

Neuroinflammation in Alzheimer's disease

(2015) Michael T Heneka et al.   LANCET NEUROLOGY

Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates

(2015) Christian Hinderer et al.   MOLECULAR THERAPY

Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates

(2015) Kathrin Meyer et al.   MOLECULAR THERAPY

Gene therapy for neurologic manifestations of mucopolysaccharidoses

(2014) Daniel A Wolf et al.   Expert Opinion on Drug Delivery

Biochemical, histological and functional correction of mucopolysaccharidosis Type IIIB by intra-cerebrospinal fluid gene therapy

(2014) Albert Ribera et al.   HUMAN MOLECULAR GENETICS

Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I

(2014) Christian Hinderer et al.   MOLECULAR THERAPY

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

(2013) S J Gray et al.   GENE THERAPY

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

(2013) Virginia Haurigot et al.   JOURNAL OF CLINICAL INVESTIGATION

Mucopolysaccharide diseases: A complex interplay between neuroinflammation, microglial activation and adaptive immunity

(2013) Louise D. Archer et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

(2012) G. Buchlis et al.   BLOOD

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

(2012) D Gaudet et al.   GENE THERAPY

Correction of Pathological Accumulation of Glycosaminoglycans in Central Nervous System and Peripheral Tissues of MPSIIIA Mice Through Systemic AAV9 Gene Transfer

(2012) Albert Ruzo et al.   HUMAN GENE THERAPY

Assessment of bone dysplasia by micro-CT and glycosaminoglycan levels in mouse models for mucopolysaccharidosis type I, IIIA, IVA, and VII

(2012) Daniel J. Rowan et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Neuropathology in Mouse Models of Mucopolysaccharidosis Type I, IIIA and IIIB

(2012) Fiona L. Wilkinson et al.   PLoS One

Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents

(2011) Roberto Calcedo et al.   Clinical and Vaccine Immunology

Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

(2011) Lluis Samaranch et al.   HUMAN GENE THERAPY

Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood–Brain Barrier Gene Delivery

(2011) Haiyan Fu et al.   MOLECULAR THERAPY

Liver Production of Sulfamidase Reverses Peripheral and Ameliorates CNS Pathology in Mucopolysaccharidosis IIIA Mice

(2011) Albert Ruzo et al.   MOLECULAR THERAPY

Ex vivo gene transfer and correction for cell-based therapies

(2011) Luigi Naldini   NATURE REVIEWS GENETICS

Inflammation in neurodegenerative diseases

(2010) Sandra Amor et al.   IMMUNOLOGY

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

(2010) Haiyan Fu et al.   JOURNAL OF GENE MEDICINE

Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations

(2010) Mark M. Souweidane et al.   Journal of Neurosurgery-Pediatrics

Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes

(2010) N Matthew Ellinwood et al.   MOLECULAR THERAPY

High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

(2009) E Ayuso et al.   GENE THERAPY

Human α-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution

(2009) Carine Ciron et al.   HUMAN GENE THERAPY

Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons

(2009) Sandra Duque et al.   MOLECULAR THERAPY

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Sanfilippo syndrome: A mini-review

(2008) M. J. Valstar et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Central nervous system therapy for lysosomal storage disorders

(2008) Gregory M. Enns et al.   Neurosurgical Focus

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE