Related references
Note: Only part of the references are listed.Biodistribution of Adeno-Associated Virus Gene Therapy Following Cerebrospinal Fluid-Directed Administration
Xin Chen et al.
HUMAN GENE THERAPY (2023)
Neurofilament light chain and dorsal root ganglia injury after adeno-associated virus 9 gene therapy in nonhuman primates
Eric W. Johnson et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2023)
Efficacy and Safety of a Krabbe Disease Gene Therapy
Juliette Hordeaux et al.
HUMAN GENE THERAPY (2022)
Characterization of AAV-mediated dorsal root ganglionopathy
Nicholas Buss et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2022)
AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates
Michael Hocquemiller et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2022)
Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy
Jonathan B. Rosenberg et al.
HUMAN GENE THERAPY (2021)
Harnessing cerebrospinal fluid circulation for drug delivery to brain tissues
Ghazal Naseri Kouzehgarani et al.
ADVANCED DRUG DELIVERY REVIEWS (2021)
Adeno-Associated Virus Vector for Central Nervous System Gene Therapy
Danqing Zhu et al.
TRENDS IN MOLECULAR MEDICINE (2021)
Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art
Maria Jose de Castro et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)
A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna
Toloo Taghian et al.
MOLECULAR THERAPY (2020)
Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
Karim Bey et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology
Juliette Hordeaux et al.
HUMAN GENE THERAPY (2020)
Adeno-associated virus serotype 1-based gene therapy for FTD caused by GRN mutations
Christian Hinderer et al.
ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY (2020)
Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2
Dolan Sondhi et al.
SCIENCE TRANSLATIONAL MEDICINE (2020)
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates
Juliette Hordeaux et al.
SCIENCE TRANSLATIONAL MEDICINE (2020)
Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality
Eloise Hudry et al.
NEURON (2019)
Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys
Juliette Hordeaux et al.
HUMAN GENE THERAPY (2019)
Mutation update: Review of TPP1 gene variants associated with neuronal ceroid lipofuscinosis CLN2 disease
Emily Gardner et al.
HUMAN MUTATION (2019)
Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates
Kousaku Ohno et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2019)
Advances in the treatment of neuronal ceroid lipofuscinosis
Jonathan B. Rosenberg et al.
EXPERT OPINION ON ORPHAN DRUGS (2019)
AAV gene delivery to the spinal cord: serotypes, methods, candidate diseases, and clinical trials
Nathan Hardcastle et al.
EXPERT OPINION ON BIOLOGICAL THERAPY (2018)
Emptying the stores: lysosomal diseases and therapeutic strategies
Frances M. Platt
NATURE REVIEWS DRUG DISCOVERY (2018)
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
Christian Hinderer et al.
HUMAN GENE THERAPY (2018)
AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease
Jonathan B. Rosenberg et al.
HUMAN GENE THERAPY CLINICAL DEVELOPMENT (2018)
Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques
Juliette Hordeaux et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)
Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study
Miriam Nickel et al.
LANCET CHILD & ADOLESCENT HEALTH (2018)
Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats
J. Hordeaux et al.
GENE THERAPY (2015)
Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates
Michel Zerah et al.
HUMAN GENE THERAPY CLINICAL DEVELOPMENT (2015)
Intracisternal delivery of AAV9 results in oligodendrocyte and Motor neuron transduction in the whole central nervous system of cats
T. Bucher et al.
GENE THERAPY (2014)
Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I
Christian Hinderer et al.
MOLECULAR THERAPY (2014)
Comparative Efficacy and Safety of Multiple Routes of Direct CNS Administration of Adeno-Associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human Arylsulfatase A cDNA to Nonhuman Primates
Jonathan B. Rosenberg et al.
HUMAN GENE THERAPY CLINICAL DEVELOPMENT (2014)
Assessment of Disease Severity in Late Infantile Neuronal Ceroid Lipofuscinosis Using Multiparametric MR Imaging
J. P. Dyke et al.
AMERICAN JOURNAL OF NEURORADIOLOGY (2013)
Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
S. J. Gray et al.
GENE THERAPY (2013)
Strong Cortical and Spinal Cord Transduction After AAV7 and AAV9 Delivery into the Cerebrospinal Fluid of Nonhuman Primates
Lluis Samaranch et al.
HUMAN GENE THERAPY (2013)
New nomenclature and classification scheme for the neuronal ceroid lipofuscinoses
Ruth E. Williams et al.
NEUROLOGY (2012)
Long-Term Expression and Safety of Administration of AAVrh.10hCLN2 to the Brain of Rats and Nonhuman Primates for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis
Dolan Sondhi et al.
HUMAN GENE THERAPY METHODS (2012)
Anatomy and physiology of cerebrospinal fluid
L. Sakka et al.
EUROPEAN ANNALS OF OTORHINOLARYNGOLOGY-HEAD AND NECK DISEASES (2011)
Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis
Dolan Sondhi et al.
EXPERIMENTAL NEUROLOGY (2008)
Neurological deterioration in late infantile neuronal ceroid lipofuscinosis
S. Worgall et al.
NEUROLOGY (2007)
Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector
Dolan Sondhi et al.
MOLECULAR THERAPY (2007)
Safety of direct administration of AAV2CUhCLN2, a candidate treatment for the central nervous system manifestations of late infantile neuronal ceroid lipofuscinosis, to the brain of rats and nonhuman primates
NR Hackett et al.
HUMAN GENE THERAPY (2005)
Feasibility of gene therapy for late neuronal ceroid lipofuscinosis
D Sondhi et al.
ARCHIVES OF NEUROLOGY (2001)
Share Paper
