Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting

Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced Pancreatitis

(2016) Daniel Gaudet et al.   HUMAN GENE THERAPY

Impact of Heparan Sulfate Binding on Transduction of Retina by Recombinant Adeno-Associated Virus Vectors

(2016) Sanford L. Boye et al.   JOURNAL OF VIROLOGY

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

(2016) Jean Bennett et al.   LANCET

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids

(2016) Kyle Chamberlain et al.   Human Gene Therapy Methods

Perfect timing: splicing and transcription rates in living cells

(2016) Tara Alpert et al.   Wiley Interdisciplinary Reviews-RNA

Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease

(2015) Ivana Trapani et al.   HUMAN MOLECULAR GENETICS

Gene therapy returns to centre stage

(2015) Luigi Naldini   NATURE

Development of an intein-mediated split–Cas9 system for gene therapy

(2015) Dong-Jiunn Jeffery Truong et al.   NUCLEIC ACIDS RESEARCH

In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector

(2015) Eric Zinn et al.   Cell Reports

The Status ofRPE65Gene Therapy Trials: Safety and Efficacy

(2015) Eric A. Pierce et al.   Cold Spring Harbor Perspectives in Medicine

Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors

(2014) P Colella et al.   GENE THERAPY

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial

(2014) Robert E MacLaren et al.   LANCET

Engineering adeno-associated viruses for clinical gene therapy

(2014) Melissa A. Kotterman et al.   NATURE REVIEWS GENETICS

Dual Adeno-Associated Virus Vectors Result in Efficient In Vitro and In Vivo Expression of an Oversized Gene, MYO7A

(2014) Frank M. Dyka et al.   Human Gene Therapy Methods

Immune Responses to AAV-Vectors, the Glybera Example from Bench to Bedside

(2014) Valerie Ferreira et al.   Frontiers in Immunology

Effective delivery of large genes to the retina by dual AAV vectors

(2013) Ivana Trapani et al.   EMBO Molecular Medicine

Regulation of Translation Initiation in Eukaryotes: Mechanisms and Biological Targets

(2009) Nahum Sonenberg et al.   CELL

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

AAV Recombineering with Single Strand Oligonucleotides

(2009) Matthew L. Hirsch et al.   PLoS One

Protein Trans-Splicing as a Means for Viral Vector-Mediated In Vivo Gene Therapy

(2008) Juan Li et al.   HUMAN GENE THERAPY

A Hybrid Vector System Expands Adeno-associated Viral Vector Packaging Capacity in a Transgene-independent Manner

(2007) Arkasubhra Ghosh et al.   MOLECULAR THERAPY