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Clinical applications of gene therapy for rare diseases: A review

International journal of experimental pathology, 2023-08, Vol.104 (4), p.154-176

2023

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Large-scale chondroitin sulfate proteoglycan digestion with chondroitinase gene therapy leads to reduced pathology and modulates macrophage phenotype following spinal cord contusion injury

The Journal of neuroscience, 2014-04, Vol.34 (14), p.4822-4836

2014

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Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice

Gene therapy, 2019-11, Vol.27 (10-11), p.505-515

2019

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Therapeutic strategies for spinal muscular atrophy: SMN and beyond

Disease models & mechanisms, 2017-08, Vol.10 (8), p.943-954

2017

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Enhanced Efficacy of the CDNF/MANF Family by Combined Intranigral Overexpression in the 6-OHDA Rat Model of Parkinson's Disease

Molecular therapy, 2015-02, Vol.23 (2), p.244-254

2015

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A study to assess a novel automated electrocardiogram technology in screening for atrial fibrillation

Pacing and clinical electrophysiology, 2019-10, Vol.42 (10), p.1383-1389

2019

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High-efficiency transduction of spinal cord motor neurons by intrauterine delivery of integration-deficient lentiviral vectors

Journal of controlled release, 2018-03, Vol.273, p.99-107

2018

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The COX‐2 inhibitors, meloxicam and nimesulide, suppress neurogenesis in the adult mouse brain

British journal of pharmacology, 2010-03, Vol.159 (5), p.1118-1125

2010

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Cortical overexpression of neuronal calcium sensor-1 induces functional plasticity in spinal cord following unilateral pyramidal tract injury in rat

PLoS biology, 2010-06, Vol.8 (6), p.e1000399

2010

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Chimeric Trojan Protein Insertion in Lentiviral Membranes Makes Lentiviruses Susceptible to Neutralization by Anti-Tetanus Serum Antibodies

Human gene therapy, 2017-03, Vol.28 (3), p.242-254

2017

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Correction: Andrew C. G. Porter (1955-2023)

Gene therapy, 2024-09, Vol.31 (9-10), p.529-529

2024

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Efficient Expression of Igf-1 from Lentiviral Vectors Protects In Vitro but Does Not Mediate Behavioral Recovery of a Parkinsonian Lesion in Rats

Human gene therapy, 2015-11, Vol.26 (11), p.719-733

2015

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Current Progress in Therapeutic Gene Editing for Monogenic Diseases

Molecular therapy, 2016-03, Vol.24 (3), p.465-474

2016

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An Induced Pluripotent Stem Cell-Derived Human Blood–Brain Barrier (BBB) Model to Test the Crossing by Adeno-Associated Virus (AAV) Vectors and Antisense Oligonucleotides

Biomedicines, 2023-10, Vol.11 (10), p.2700

2023

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Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models

Gene therapy, 2022-09, Vol.29 (9), p.498-512

2022

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CAR-T in the clinic: drive with care

Gene therapy, 2018-06, Vol.25 (3), p.157-161

2018

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Gene therapy for spinal muscular atrophy: perspectives on the possibility of optimizing SMN1 delivery to correct all neurological and systemic perturbations

Neural regeneration research, 2025-07, Vol.20 (7), p.2011-2012

2025

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Lentiviral vectors encoding short hairpin RNAs efficiently transduce and knockdown LINGO-1 but induce an interferon response and cytotoxicity in central nervous system neurones

The journal of gene medicine, 2012-05, Vol.14 (5), p.299-315

2012

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Activation of EphA Receptors Mediates the Recruitment of the Adaptor Protein Slap, Contributing to the Downregulation of N-Methyl-d-Aspartate Receptors

Molecular and Cellular Biology, 2013-04, Vol.33 (7), p.1442-1455

2013

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Genomic insertion of lentiviral DNA circles directed by the yeast Flp recombinase

BMC biotechnology, 2008-08, Vol.8 (1), p.60-60, Article 60

2008

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Highly potent delivery method of gp160 envelope vaccine combining lentivirus-like particles and DNA electrotransfer

Journal of controlled release, 2012-05, Vol.159 (3), p.376-383

2012

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Induction of the cell survival kinase Sgk1: A possible novel mechanism for α-phenyl-N-tert-butyl nitrone in experimental stroke

Journal of cerebral blood flow and metabolism, 2019-06, Vol.39 (6), p.1111-1121

2019

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Decay in survival motor neuron and plastin 3 levels during differentiation of iPSC-derived human motor neurons

Scientific reports, 2015-06, Vol.5 (1), p.11696-11696, Article 11696

2015

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Rapid high-resolution karyotyping with precise identification of chromosome breakpoints

Genes chromosomes & cancer, 2007-07, Vol.46 (7), p.675-683

2007

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Clinical applications of gene therapy for rare diseases: A review

Large-scale chondroitin sulfate proteoglycan digestion with chondroitinase gene therapy leads to reduced pathology and modulates macrophage phenotype following spinal cord contusion injury

Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice

Therapeutic strategies for spinal muscular atrophy: SMN and beyond

Enhanced Efficacy of the CDNF/MANF Family by Combined Intranigral Overexpression in the 6-OHDA Rat Model of Parkinson's Disease

A study to assess a novel automated electrocardiogram technology in screening for atrial fibrillation

High-efficiency transduction of spinal cord motor neurons by intrauterine delivery of integration-deficient lentiviral vectors

The COX‐2 inhibitors, meloxicam and nimesulide, suppress neurogenesis in the adult mouse brain

Cortical overexpression of neuronal calcium sensor-1 induces functional plasticity in spinal cord following unilateral pyramidal tract injury in rat

Chimeric Trojan Protein Insertion in Lentiviral Membranes Makes Lentiviruses Susceptible to Neutralization by Anti-Tetanus Serum Antibodies

Correction: Andrew C. G. Porter (1955-2023)

Efficient Expression of Igf-1 from Lentiviral Vectors Protects In Vitro but Does Not Mediate Behavioral Recovery of a Parkinsonian Lesion in Rats

Current Progress in Therapeutic Gene Editing for Monogenic Diseases

An Induced Pluripotent Stem Cell-Derived Human Blood–Brain Barrier (BBB) Model to Test the Crossing by Adeno-Associated Virus (AAV) Vectors and Antisense Oligonucleotides

Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models

CAR-T in the clinic: drive with care

Gene therapy for spinal muscular atrophy: perspectives on the possibility of optimizing SMN1 delivery to correct all neurological and systemic perturbations

Lentiviral vectors encoding short hairpin RNAs efficiently transduce and knockdown LINGO-1 but induce an interferon response and cytotoxicity in central nervous system neurones

Activation of EphA Receptors Mediates the Recruitment of the Adaptor Protein Slap, Contributing to the Downregulation of N-Methyl-d-Aspartate Receptors

Genomic insertion of lentiviral DNA circles directed by the yeast Flp recombinase

Highly potent delivery method of gp160 envelope vaccine combining lentivirus-like particles and DNA electrotransfer

Induction of the cell survival kinase Sgk1: A possible novel mechanism for α-phenyl-N-tert-butyl nitrone in experimental stroke

Decay in survival motor neuron and plastin 3 levels during differentiation of iPSC-derived human motor neurons

Comprehensive genomic access to vector integration in clinical gene therapy

Rapid high-resolution karyotyping with precise identification of chromosome breakpoints

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