The principles of gene therapy for the nervous system

Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis

1993 • 6,762 citations

Identification and characterization of a spinal muscular atrophy-determining gene

1995 • 3,722 citations

The Basic Science of Gene Therapy

1993 • 1,767 citations

Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection.

1990 • 1,465 citations

Antisense Oligonucleotides as Therapeutic Agents—Is the Bullet Really Magical?

1993 • 1,306 citations

HPRT-deficient (Lesch–Nyhan) mouse embryos derived from germline colonization by cultured cells

1987 • 1,231 citations

Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain

1994 • 1,032 citations

The gene for neuronal apoptosis inhibitory protein is partially deleted in individuals with spinal muscular atrophy

1995 • 976 citations

Isolation, Characterization, and use of Stem Cells from the CNS

1995 • 819 citations

An Adenovirus Vector for Gene Transfer into Neurons and Glia in the Brain

1993 • 746 citations

Transfer of a foreign gene into the brain using adenovirus vectors

1993 • 538 citations

A potential animal model for Lesch–Nyhan syndrome through introduction of HPRT mutations into mice

1987 • 487 citations

GFAP promoter directs astrocyte-specific expression in transgenic mice

1994 • 471 citations

Neuropathological changes in two lines of mice carrying a transgene for mutant human Cu,Zn SOD, and in mice overexpressing wild type human SOD: a model of familial amyotrophic lateral sclerosis (FALS)

1995 • 435 citations

PDGF B-chain in neurons of the central nervous system, posterior pituitary, and in a transgenic model

1991 • 410 citations

Long-Term Behavioral Recovery in Parkinsonian Rats by an HSV Vector Expressing Tyrosine Hydroxylase

1994 • 333 citations

Adenovirus gene transfer causes inflammation in the brain

1995 • 283 citations

Gene therapy for cancer

1994 • 244 citations

Myelin acquisition in the central nervous system of the mouse revealed by an MBP-Lac Z transgene

1992 • 239 citations

Neuronal transfection in brain slices using particle-mediated gene transfer

1994 • 237 citations

The T alpha 1 alpha-tubulin promoter specifies gene expression as a function of neuronal growth and regeneration in transgenic mice

1994 • 196 citations

Transneuronal Transfer of Herpes Virus from Peripheral Nerves to Cortex and Brainstem

1989 • 175 citations

Direct intracerebral gene transfer of an adenoviral vector expressing tyrosine hydroxylase in a rat model of Parkinsonʼs disease

1994 • 160 citations

Expression of human HPRT mRNA in brains of mice infected with a recombinant herpes simplex virus-1 vector

1989 • 127 citations

CNS gene delivery by retrograde transport of recombinant replication-defective adenoviruses.

1995 • 125 citations

Persistence of plasmid DNA and expression in rat brain cells in vivo

1992 • 119 citations

Gene transfer and expression in progeny after intravenous DNA injection into pregnant mice

1995 • 118 citations

Grafting in the mammalian central nervous system

1993 • 114 citations

Gene Therapy for Neurologic Disease

1993 • 113 citations

Gene Expression from Recombinant Viral Vectors in the Central Nervous System after Blood-Brain Barrier Disruption

1995 • 107 citations

Gene Therapy for the Treatment of Malignant Brain Tumors with In Vivo Tumor Transduction with the Herpes Simplex Thymidine Kinase Gene/Ganciclovir System. Iowa Methodist Medical Center, Des Moines, Iowa

1994 • 104 citations

5' flanking sequences of the rat tyrosine hydroxylase gene target accurate tissue-specific, developmental, and transsynaptic expression in transgenic mice

1992 • 99 citations

Molecular analysis of neuronal physiology by gene transfer into neurons with herpes simplex virus vectors

1991 • 98 citations

Gene delivery into the central nervous system by nasal instillation in rats.

1995 • 84 citations

Adenoviral vectors as functional retrograde neuronal tracers

1994 • 74 citations

Gene therapy for neurological disorders

1994 • 73 citations

Herpesvirus-mediated gene delivery into the rat brain: specificity and efficiency of the neuron-specific enolase promoter

1993 • 70 citations

Adenovirus‐mediated gene transfer to murine retinal cells in vitro and in vivo

1994 • 64 citations

Transgenic and Knock‐out Mice: Models of Neurological Disease

1994 • 63 citations

Gene transfer to brain using herpes simplex virus vectors

1994 • 63 citations

Adenovirus vectors enter the brain

1993 • 60 citations

Herpes simplex virus vector system: analysis of its in vivo and in vitro cytopathic effects

1995 • 56 citations

Transfer of the nerve growth factor gene into cell lines and cultured neurons using a defective Herpes Simplex virus vector. Transfer of the NGF gene into cells by a HSV-1 vector

1994 • 53 citations

Gene therapy for mitochondrial DNA defects: is it possible?

1995 • 51 citations

Intragenic regulatory elements contribute to transcriptional control of the neurofilament light gene

1992 • 50 citations

Molecular Basis of Common Hereditary Motor and Sensory Neuropathies in Humans and in Mouse Models

1995 • 50 citations

Astrogliosis in culture: III. Effect of recombinant retrovirus expressing antisense glial fibrillary acidic protein RNA

1994 • 48 citations

High‐Efficiency Gene Transfer Mediated by Adenovirus‐Polylysine‐DNA Complexes

1994 • 41 citations

In vivo and ex vivo gene transfer to the brain

1994 • 37 citations

Transfecting neurons and glia in the rat using pH-sensitive immunoliposomes

1995 • 29 citations

Gene delivery to spinal motor neurons

1993 • 25 citations

In vivo transfer of a marker gene to study motoneuronal development

1994 • 23 citations

The use of herpes simplex virus vectors for gene therapy in neurological diseases

1993 • 22 citations

Gene therapy and the brain

1995 • 19 citations

Sustained expression of functional nerve growth factor in primary septo-hippocampal cell cultures by liposome-mediated gene transfer

1994 • 15 citations

Gene transfer and the expression of a foreign gene in vivo in post-mitotic neurons of the adult rat brain using the hemagglutinating virus of the Japan-liposome method

1994 • 14 citations

Inherited Neuropathies: The Interface between Molecular Genetics and Pathology

1993 • 14 citations

Postnatal gene transfer into the central nervous system

1993 • 13 citations

Gene targeting and synaptic plasticity

1994 • 10 citations