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Gene therapy corrects the neurological deficits of mice with sialidosis
- Wuh-Liang Hwu
- , Karine Chang
- & Yin-Hsiu Chien
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AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes
- Brahim Chaqour
- , Thu T. Duong
- & Ahmara G. Ross
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CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy
- Emrah Gumusgoz
- , Sahba Kasiri
- & Berge A. Minassian
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Brief Communication
| Open Access
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver
- Carola J. Maturana
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Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability
- Evgeny S. Nikitin
- , Tatiana Y. Postnikova
- & Aleksey V. Zaitsev
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Article
| Open AccessSuccesses and challenges in clinical gene therapy
- Donald B. Kohn
- , Yvonne Y. Chen
- & Melissa J. Spencer
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Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo
- Gabriela Toro Cabrera
- , Katharina E. Meijboom
- & Christian Mueller
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Article
| Open Access
CRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´s disease
- Luis Fernando Narváez-Pérez
- , Francisco Paz-Bermúdez
- & José Segovia
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Article
| Open Access
Combinatorial gene therapy for epilepsy: Gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures
- Esbjörn Melin
- , My Andersson
- & Merab Kokaia
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Novel rAAV vector mediated intrathecal HGF delivery has an impact on neuroimmune modulation in the ALS motor cortex with TDP-43 pathology
- Barış Genç
- , Boram Nho
- & Hande Özdinler
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Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9
- Pouiré Yaméogo
- , Catherine Gérard
- & Jacques P. Tremblay
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Brief Communication |
Ptbp1 knockdown failed to induce astrocytes to neurons in vivo
- Guixiang Yang
- , Zixiang Yan
- & Kailun Fang
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Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes
- Jayanth Chandran
- , Ekram Ahmed Chowdhury
- & Guy M. L. Meno-Tetang
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Brief Communication
| Open Access
Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study
- Ilaria Bitetti
- , Valentina Lanzara
- & Antonio Varone
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CRISPR/Cas9-based genome editing for the modification of multiple duplications that cause Duchenne muscular dystrophy
- Dan-Ni Wang
- , Zhi-Qiang Wang
- & Ning Wang
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Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence
- Sharon Aharoni
- , Jacob Bistritzer
- & Yoram Nevo
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Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy
- Aoife Reilly
- , Marc-Olivier Deguise
- & Rashmi Kothary
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Comment |
Friend or Foe(tal): challenges in development of a large animal model for pre-clinical fetal gene therapy
- Richard S. Finkel
- & Christian L. Lorson
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Article
| Open Access
Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1
- Siham Ait Benichou
- , Dominic Jauvin
- & Jack Puymirat
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Upper motor neurons are a target for gene therapy and UCHL1 is necessary and sufficient to improve cellular integrity of diseased upper motor neurons
- Barış Genç
- , Javier H. Jara
- & P. Hande Özdinler
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Outer retinal transduction by AAV2-7m8 following intravitreal injection in a sheep model of CNGA3 achromatopsia
- M. Ross
- , A. Obolensky
- & R. Ofri
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Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets
- Kelly A. Rich
- , Christopher G. Wier
- & Stephen J. Kolb
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Article
| Open Access
Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy
- Vahid Farrokhi
- , Jason Walsh
- & Hendrik Neubert
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Injection pressure levels for creating blebs during subretinal gene therapy
- Brittni A. Scruggs
- , Huber Martins Vasconcelos Jr.
- & Andreas K. Lauer
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Article
| Open Access
Rs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation
- Yong Zeng
- , Haohua Qian
- & Paul A. Sieving
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Article
| Open Access
Development and biological characterization of a clinical gene transfer vector for the treatment of MAK-associated retinitis pigmentosa
- Budd A. Tucker
- , Erin R. Burnight
- & Edwin M. Stone
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CCR1 enhances SUMOylation of DGCR8 by up-regulating ERK phosphorylation to promote spinal nerve ligation-induced neuropathic pain
- Cunxian Shi
- , Jin Jin
- & Zhen Li
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Article
| Open Access
Gene therapy for spinal muscular atrophy: the Qatari experience
- Hossamaldein Gaber Ali
- , Khalid Ibrahim
- & Tawfeg Ben-Omran
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Brief Communication
| Open Access
Advantages of robotic assistance over a manual approach in simulated subretinal injections and its relevance for gene therapy
- Reza Ladha
- , Thijs Meenink
- & Marc D. de Smet
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Striatal ΔFosB gene suppression inhibits the development of abnormal involuntary movements induced by L-Dopa in rats
- Goichi Beck
- , Jie Zhang
- & Stella M. Papa
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Article
| Open Access
AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy
- Alexia Kagiava
- , Christos Karaiskos
- & Kleopas A. Kleopa
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Proteosomal degradation impairs transcytosis of AAV vectors from suprachoroidal space to retina
- Kun Ding
- , Jikui Shen
- & Peter A. Campochiaro
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Article
| Open Access
AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1
- Burcak Ozes
- , Morgan Myers
- & Zarife Sahenk
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Review Article
| Open Access
Genome editing for Duchenne muscular dystrophy: a glimpse of the future?
- Christian Kupatt
- , Alina Windisch
- & Maggie C. Walter
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Article
| Open Access
Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells
- Andrea J. Korecki
- , Jorge L. Cueva-Vargas
- & Elizabeth M. Simpson
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Brief Communication
| Open Access
Single-cell mapping of focused ultrasound-transfected brain
- A. S. Mathew
- , C. M. Gorick
- & R. J. Price
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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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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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Review Article |
Gene therapeutic strategies and relevant clinical trials in neuromuscular disorder in China
- Chaoping Hu
- & Xihua Li
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Comment |
Retinal gene therapy: an eye-opener of the 21st century
- Anne Louise Askou
- , Thomas Stax Jakobsen
- & Thomas J. Corydon
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Review Article |
Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy
- Kasia Dzierlega
- & Toshifumi Yokota
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Review Article |
Reactivation of FMR1 gene expression is a promising strategy for fragile X syndrome therapy
- Ekaterina M. Shitik
- , Anastasia A. Velmiskina
- & Dmitry V. Yudkin
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Adeno-associated virus vector enables safe and efficient Cas9 activation in neonatal and adult Cas9 knockin murine cochleae
- Wen Kang
- , Xingle Zhao
- & Hao Wu
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A novel gene editing system to treat both Tay–Sachs and Sandhoff diseases
- Li Ou
- , Michael J. Przybilla
- & Chester B. Whitley
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The deletion of mutant SOD1 via CRISPR/Cas9/sgRNA prolongs survival in an amyotrophic lateral sclerosis mouse model
- Weisong Duan
- , Moran Guo
- & Chunyan Li
Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease
Curing SMA: Are we there yet?
CRISPR editing as a therapeutic strategy for Duchenne muscular dystrophy—anti-Cas9 immune response casts its shadow over safety and efficacyBrowse narrower subjects
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