Featured
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Review Article |
CRISPR/Cas9-mediated base editors and their prospects for mitochondrial genome engineering
- Shahin Eghbalsaied
- , Clancy Lawler
- & Stephen Frankenberg
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Brief Communication |
Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement
- A. Mateu-Bosch
- , E. Segur-Bailach
- & C. Fillat
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Article |
Long-term effects of a fat-directed FGF21 gene therapy in aged female mice
- Jacqueline M. Anderson
- , W. David Arnold
- & Lei Cao
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Comment |
Comment on: Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets
- Apeksha Dave
- , Cara L. Berkowitz
- & William H. Peranteau
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Article
| Open Access
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
- Jey M. Jeyakumar
- , Azadeh Kia
- & Amit Nathwani
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Article |
Activation of homology-directed DNA repair plays key role in CRISPR-mediated genome correction
- Gourish Mondal
- , Caitlin J. VanLith
- & Joseph B. Lillegard
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Article |
IgG-cleavage protein allows therapeutic AAV gene delivery in passively immunized MPS IIIA mice
- Tierra A. Bobo
- , Preston N. Samowitz
- & Haiyan Fu
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Article |
A novel preclinical model of mucopolysaccharidosis type II for developing human hematopoietic stem cell gene therapy
- Yohta Shimada
- , Natsumi Ishii
- & Hiroshi Kobayashi
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Article |
Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model
- Jie Gong
- , Yunyun Liu
- & Lung-Ji Chang
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Article |
Efficient CRISPR/Cas9 nickase-mediated genome editing in an in vitro model of mucopolysaccharidosis IVA
- Andrés Felipe Leal
- & Carlos Javier Alméciga-Díaz
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Article
| Open Access
Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse
- Janine A. Gilkes
- , Benjamin L. Judkins
- & Coy D. Heldermon
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Article |
Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease
- Victoria J. McCurdy
- , Aime K. Johnson
- & Douglas R. Martin
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Article |
Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice
- Roselena Silvestri Schuh
- , Esteban Alberto Gonzalez
- & Guilherme Baldo
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Brief Communication |
Design and applications of gene therapy vectors for mucopolysaccharidosis in Colombia
- Carlos J. Alméciga-Diaz
- & Luis A. Barrera
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Article |
Ectopic BAT mUCP-1 overexpression in SKM by delivering a BMP7/PRDM16/PGC-1a gene cocktail or single PRMD16 using non-viral UTMD gene therapy
- Shuyuan Chen
- , Raul A. Bastarrachea
- & Paul A. Grayburn
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Brief Communication |
HIV-based lentivirus-mediated vasoactive intestinal peptide gene delivery protects against DIO animal model of Type 2 diabetes
- Hale M. Tasyurek
- , Yunus E. Eksi
- & Salih Sanlioglu
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Original Article |
Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler–Najjar Syndrome Type I
- L Bočkor
- , G Bortolussi
- & A F Muro
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Short Communication |
Gene therapy for spinomuscular atrophy: a biomedical advance, a missed opportunity for more equitable drug pricing
- T Friedmann
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Comment |
Delivering efficient liver-directed AAV-mediated gene therapy
- J Baruteau
- , S N Waddington
- & P Gissen
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Original Article |
Adeno-associated virus-mediated expression of myostatin propeptide improves the growth of skeletal muscle and attenuates hyperglycemia in db/db mice
- J G Jiang
- , G F Shen
- & X Xiao
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Original Article |
Interferon beta overexpression attenuates adipose tissue inflammation and high-fat diet-induced obesity and maintains glucose homeostasis
- M Alsaggar
- , M Mills
- & D Liu
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Original Article |
Long-term, high-level hepatic secretion of acid α-glucosidase for Pompe disease achieved in non-human primates using helper-dependent adenovirus
- D P W Rastall
- , S S Seregin
- & A Amalfitano
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Original Article |
Construction of minicircle DNA vectors capable of correcting familial hypercholesterolemia phenotype in a LDLR-deficient mouse model
- X Hou
- , R Jiao
- & Z-Y Chen
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Original Article |
IL-15/sIL-15Rα gene transfer induces weight loss and improves glucose homeostasis in obese mice
- H Sun
- , Y Ma
- & D Liu
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Original Article |
Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1
- R Castello
- , R Borzone
- & N Brunetti-Pierri
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Original Article |
Helper-dependent adenovirus achieve more efficient and persistent liver transgene expression in non-human primates under immunosuppression
- C Unzu
- , I Melero
- & A Fontanellas
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Original Article |
Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease
- V J McCurdy
- , H E Rockwell
- & D R Martin
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Short Communication |
AAVrh.10 immunogenicity in mice and humans. Relevance of antibody cross-reactivity in human gene therapy
- R Thwaite
- , G Pagès
- & A Bosch
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Original Article |
High and prolonged sulfamidase secretion by the liver of MPS-IIIA mice following hydrodynamic tail vein delivery of antibiotic-free pFAR4 plasmid vector
- M Quiviger
- , A Arfi
- & C Marie
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Original Article |
Overexpression of superoxide dismutase 3 gene blocks high-fat diet-induced obesity, fatty liver and insulin resistance
- R Cui
- , M Gao
- & D Liu
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Original Article |
Thymidine phosphorylase is both a therapeutic and a suicide gene in a murine model of mitochondrial neurogastrointestinal encephalomyopathy
- S López-Estévez
- , G Ferrer
- & J Barquinero
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Short Communication |
Variable correction of Artemis deficiency by I-Sce1-meganuclease-assisted homologous recombination in murine hematopoietic stem cells
- J Rivière
- , J Hauer
- & M Cavazzana
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Short Communication |
Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1
- R J Chandler
- , T N Tarasenko
- & P J McGuire
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Short Communication |
AAV-encoded OTC activity persisting to adulthood following delivery to newborn spfash mice is insufficient to prevent shRNA-induced hyperammonaemia
- S C Cunningham
- , C Y Kok
- & I E Alexander
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Original Article |
Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice
- C D Heldermon
- , E Y Qin
- & M S Sands
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Original Article |
AAV-based gene therapy prevents neuropathology and results in normal cognitive development in the hyperargininemic mouse
- E K Lee
- , C Hu
- & G S Lipshutz
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Original Article |
Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
- S J Gray
- , S Nagabhushan Kalburgi
- & R Jude Samulski
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Original Article |
Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial
- D Gaudet
- , J Méthot
- & S van Deventer
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Original Article |
Manipulation of mtDNA heteroplasmy in all striated muscles of newborn mice by AAV9-mediated delivery of a mitochondria-targeted restriction endonuclease
- S R Bacman
- , S L Williams
- & C T Moraes
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Original Article |
Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease
- B Sun
- , S Li
- & D D Koeberl
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News and Commentary |
The battle to prevent the transmission of mitochondrial DNA disease: Is karyoplast transfer the answer?
- J C St John
- & K H S Campbell
Gene therapy corrects the neurological deficits of mice with sialidosis
Hydrodynamic delivery of interleukin 15 gene promotes resistance to high fat diet-induced obesity, fatty liver and improves glucose homeostasis