Advances in AAV vector development for gene therapy in the retina.
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Lipid Nanoparticles for Ocular Gene DeliveryLigand-binding domains of nuclear receptors facilitate tight control of split CRISPR activityMassively parallel cis-regulatory analysis in the mammalian central nervous systemImmobilization of FLAG-Tagged Recombinant Adeno-Associated Virus 2 onto Tissue Engineering Scaffolds for the Improvement of Transgene Delivery in Cell Transplants.Bulk regional viral injection in neonatal mice enables structural and functional interrogation of defined neuronal populations throughout targeted brain areas.Tyrosine-mutated AAV2-mediated shRNA silencing of PTEN promotes axon regeneration of adult optic nerveCrxRdy Cat: A Large Animal Model for CRX-Associated Leber Congenital Amaurosis.Retinal Structure and Gene Therapy Outcome in Retinoschisin-Deficient Mice Assessed by Spectral-Domain Optical Coherence Tomography.Recombinant adeno-associated virus vectors in the treatment of rare diseases.Gene therapy for age-related macular degeneration.Report on the National Eye Institute Audacious Goals Initiative: Replacement of Retinal Ganglion Cells from Endogenous Cell Sources.Beyond traditional pharmacology: new tools and approaches.The baculovirus expression vector system: A commercial manufacturing platform for viral vaccines and gene therapy vectors.Applications of CRISPR/Cas9 in retinal degenerative diseasesAn optimized gene transfection system in WERI-Rb1 cells.miRNA-mediated post-transcriptional silencing of transgenes leads to increased adeno-associated viral vector yield and targeting specificity.Concepts and Strategies in Retinal Gene Therapy.Gene therapy for inherited retinal and optic nerve degenerations.Methods for In Vivo CRISPR/Cas Editing of the Adult Murine Retina.Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy.
P2860
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P2860
Advances in AAV vector development for gene therapy in the retina.
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article científic
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article scientifique
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articol științific
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articolo scientifico
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artigo científico
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artigo científico
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artigo científico
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Advances in AAV vector development for gene therapy in the retina.
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type
label
Advances in AAV vector development for gene therapy in the retina.
@en
prefLabel
Advances in AAV vector development for gene therapy in the retina.
@en
P2860
P1476
Advances in AAV vector development for gene therapy in the retina.
@en
P2093
Leah C Byrne
Timothy P Day
P2860
P304
P356
10.1007/978-1-4614-3209-8_86
P407
P577
2014-01-01T00:00:00Z