AAV-Mediated Gene Supplementation Therapy in Achromatopsia Type 2: Preclinical Data on Therapeutic Time Window and Long-Term Effects
出版年份 2017 全文链接
标题
AAV-Mediated Gene Supplementation Therapy in Achromatopsia Type 2: Preclinical Data on Therapeutic Time Window and Long-Term Effects
作者
关键词
-
出版物
Frontiers in Neuroscience
Volume 11, Issue -, Pages -
出版商
Frontiers Media SA
发表日期
2017-05-24
DOI
10.3389/fnins.2017.00292
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Gene therapy for achromatopsia
- (2017) Stylianos Michalakis et al. JOURNAL OF GENE MEDICINE
- Advances in Gene Therapy for Diseases of the Eye
- (2016) Lolita Petit et al. HUMAN GENE THERAPY
- Men and mice: Relating their ages
- (2016) Sulagna Dutta et al. LIFE SCIENCES
- Emerging therapies for inherited retinal degeneration
- (2016) H. P. N. Scholl et al. Science Translational Medicine
- AAV Vectors for FRET-Based Analysis of Protein-Protein Interactions in Photoreceptor Outer Segments
- (2016) Elvir Becirovic et al. Frontiers in Neuroscience
- The cone dysfunction syndromes: Table 1
- (2015) Jonathan Aboshiha et al. BRITISH JOURNAL OF OPHTHALMOLOGY
- Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia
- (2015) Eyal Banin et al. MOLECULAR THERAPY
- Mutations in the unfolded protein response regulator ATF6 cause the cone dysfunction disorder achromatopsia
- (2015) Susanne Kohl et al. NATURE GENETICS
- Achromatopsia: On the Doorstep of a Possible Therapy
- (2015) Ditta Zobor et al. OPHTHALMIC RESEARCH
- Scale Adjustments to Facilitate Two-Dimensional Measurements in OCT Images
- (2015) Marina Garcia Garrido et al. PLoS One
- Identification of a Common Non-Apoptotic Cell Death Mechanism in Hereditary Retinal Degeneration
- (2014) Blanca Arango-Gonzalez et al. PLoS One
- Combined Rod and Cone Transduction by Adeno-Associated Virus 2/8
- (2013) Anna Manfredi et al. HUMAN GENE THERAPY
- Gene replacement therapy for retinal CNG channelopathies
- (2013) Christian Schön et al. MOLECULAR GENETICS AND GENOMICS
- Transient Photoreceptor Deconstruction by CNTF Enhances rAAV-Mediated Cone Functional Rescue in Late Stage CNGB3-Achromatopsia
- (2013) András M Komáromy et al. MOLECULAR THERAPY
- AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina
- (2013) Luk H. Vandenberghe et al. PLoS One
- A Nonsense Mutation in PDE6H Causes Autosomal-Recessive Incomplete Achromatopsia
- (2012) Susanne Kohl et al. AMERICAN JOURNAL OF HUMAN GENETICS
- Gene therapy restores vision and delays degeneration in the CNGB1-/- mouse model of retinitis pigmentosa
- (2012) S. Koch et al. HUMAN MOLECULAR GENETICS
- AAV-Mediated Cone Rescue in a Naturally Occurring Mouse Model of CNGA3-Achromatopsia
- (2012) Ji-jing Pang et al. PLoS One
- Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy
- (2011) L. S. Carvalho et al. HUMAN MOLECULAR GENETICS
- Molecular mechanism for 3:1 subunit stoichiometry of rod cyclic nucleotide-gated ion channels
- (2011) Noah G. Shuart et al. Nature Communications
- Gene therapy rescues cone function in congenital achromatopsia
- (2010) András M. Komáromy et al. HUMAN MOLECULAR GENETICS
- Restoration of Cone Vision in the CNGA3−/− Mouse Model of Congenital Complete Lack of Cone Photoreceptor Function
- (2010) Stylianos Michalakis et al. MOLECULAR THERAPY
- Long-term Retinal Function and Structure Rescue Using Capsid Mutant AAV8 Vector in the rd10 Mouse, a Model of Recessive Retinitis Pigmentosa
- (2010) Ji-jing Pang et al. MOLECULAR THERAPY
- Achromatopsie
- (2010) C.M. Poloschek et al. OPHTHALMOLOGE
- Homozygosity Mapping Reveals PDE6C Mutations in Patients with Early-Onset Cone Photoreceptor Disorders
- (2009) Alberta A.H.J. Thiadens et al. AMERICAN JOURNAL OF HUMAN GENETICS
- Vision tests in the mouse: Functional phenotyping with electroretinography
- (2009) Naoyuki Tanimoto Frontiers in Bioscience-Landmark
- Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints
- (2009) L H Vandenberghe et al. GENE THERAPY
- Gene therapy for red–green colour blindness in adult primates
- (2009) Katherine Mancuso et al. NATURE
- Noninvasive, In Vivo Assessment of Mouse Retinal Structure Using Optical Coherence Tomography
- (2009) M. Dominik Fischer et al. PLoS One
- A homologous genetic basis of the murine cpfl1 mutant and human achromatopsia linked to mutations in the PDE6C gene
- (2009) B. Chang et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- High-efficiency Transduction of the Mouse Retina by Tyrosine-mutant AAV Serotype Vectors
- (2008) Hilda Petrs-Silva et al. MOLECULAR THERAPY
- Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression
- (2008) Li Zhong et al. VIROLOGY
Create your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create NowBecome a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get Started