Human stem cell-based retina on chip as new translational model for validation of AAV retinal gene therapy vectors

Gene Therapy of Dominant CRX-Leber Congenital Amaurosis using Patient Stem Cell-Derived Retinal Organoids

(2021) Kamil Kruczek et al.   Stem Cell Reports

Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders

(2021) Marina Pavlou et al.   EMBO Molecular Medicine

AAV-Mediated Gene Delivery to 3D Retinal Organoids Derived from Human Induced Pluripotent Stem Cells

(2020) Marcela Garita-Hernandez et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

A Small-Molecule-Responsive Riboswitch Enables Conditional Induction of Viral Vector-Mediated Gene Expression in Mice

(2020) Benjamin Strobel et al.   ACS Synthetic Biology

Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays

(2020) Thilo Buck et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

In vivo–directed evolution of adeno-associated virus in the primate retina

(2020) Leah C. Byrne et al.   JCI Insight

Cell Types of the Human Retina and Its Organoids at Single-Cell Resolution

(2020) Cameron S. Cowan et al.   CELL

Organs-on-chips: into the next decade

(2020) Lucie A. Low et al.   NATURE REVIEWS DRUG DISCOVERY

Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors

(2019) Hildegard Büning et al.   Molecular Therapy-Methods & Clinical Development

Standardized, Scalable, and Timely Flexible Adeno-Associated Virus Vector Production Using Frozen High-Density HEK-293 Cell Stocks and CELLdiscs

(2019) Benjamin Strobel et al.   Human Gene Therapy Methods

Impact of organ-on-a-chip technology on pharmaceutical R&D costs

(2019) Nora Franzen et al.   DRUG DISCOVERY TODAY

Merging organoid and organ-on-a-chip technology to generate complex multi-layer tissue models in a human retina-on-a-chip platform

(2019) Kevin Achberger et al.   eLife

Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders

(2019) Alireza Shahryari et al.   Frontiers in Genetics

Progress in treating inherited retinal diseases: Early subretinal gene therapy clinical trials and candidates for future initiatives

(2019) Alexandra V. Garafalo et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Assessment of Adeno-Associated Virus Serotype Tropism in Human Retinal Explants

(2018) Luke A. Wiley et al.   HUMAN GENE THERAPY

Assessment of AAV Vector Tropisms for Mouse and Human Pluripotent Stem Cell–Derived RPE and Photoreceptor Cells

(2018) Anai Gonzalez-Cordero et al.   HUMAN GENE THERAPY

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Seeing the Light after 25 Years of Retinal Gene Therapy

(2018) Ivana Trapani et al.   TRENDS IN MOLECULAR MEDICINE

Advances in organ-on-a-chip engineering

(2018) Boyang Zhang et al.   Nature Reviews Materials

Organ-on-a-chip technologies that can transform ophthalmic drug discovery and disease modeling

(2018) Jasmin C. Haderspeck et al.   Expert Opinion on Drug Discovery

Preclinical Evaluation of ADVM-022, a Novel Gene Therapy Approach to Treating Wet Age-Related Macular Degeneration

(2018) Ruslan Grishanin et al.   MOLECULAR THERAPY

Adeno-Associated Viral Vector 2 and 9 Transduction Is Enhanced in Streptozotocin-Induced Diabetic Mouse Retina

(2018) Si Hyung Lee et al.   Molecular Therapy-Methods & Clinical Development

Host determinants of adeno-associated viral vector entry

(2017) Sirika Pillay et al.   Current Opinion in Virology

Retinal Gene Therapy: Surgical Vector Delivery in the Translation to Clinical Trials

(2017) G. Alex Ochakovski et al.   Frontiers in Neuroscience

Insight into the mechanisms of enhanced retinal transduction by the engineered AAV2 capsid variant -7m8

(2016) Hanen Khabou et al.   BIOTECHNOLOGY AND BIOENGINEERING

High-Efficiency Transduction of Primary Human Hematopoietic Stem/Progenitor Cells by AAV6 Vectors: Strategies for Overcoming Donor-Variation and Implications in Genome Editing

(2016) Chen Ling et al.   Scientific Reports

Comparative Analysis of Cesium Chloride- and Iodixanol-Based Purification of Recombinant Adeno-Associated Viral Vectors for Preclinical Applications

(2015) Benjamin Strobel et al.   Human Gene Therapy Methods

Generation of three-dimensional retinal tissue with functional photoreceptors from human iPSCs

(2014) Xiufeng Zhong et al.   Nature Communications

In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

(2013) D. Dalkara et al.   Science Translational Medicine

Small Molecule-Assisted, Line-Independent Maintenance of Human Pluripotent Stem Cells in Defined Conditions

(2012) Stefan Frank et al.   PLoS One

Clinical applications of retinal gene therapy

(2012) Daniel M. Lipinski et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Lentiviral Vector Design and Imaging Approaches to Visualize the Early Stages of Cellular Reprogramming

(2011) Eva Warlich et al.   MOLECULAR THERAPY

Rat Embryonic Fibroblasts Improve Reprogramming of Human Keratinocytes into Induced Pluripotent Stem Cells

(2011) Leonhard Linta et al.   STEM CELLS AND DEVELOPMENT

A Novel Adeno-Associated Viral Variant for Efficient and Selective Intravitreal Transduction of Rat Müller Cells

(2009) Ryan R. Klimczak et al.   PLoS One