Formulation and efficacy of ECO/pRHO-ABCA4-SV40 nanoparticles for nonviral gene therapy of Stargardt disease in a mouse model

Polyester based polymeric nano and microparticles for pharmaceutical purposes: A review on formulation approaches

(2020) Fatima Molavi et al.   JOURNAL OF CONTROLLED RELEASE

Gene therapy beyond luxturna

(2020) Dominic A. Prado et al.   CURRENT OPINION IN OPHTHALMOLOGY

Systemic Delivery of Tumor-Targeting siRNA Nanoparticles against an Oncogenic LncRNA Facilitates Effective Triple-Negative Breast Cancer Therapy

(2019) Amita M Vaidya et al.   BIOCONJUGATE CHEMISTRY

Formulation of Biocompatible Targeted ECO/siRNA Nanoparticles with Long-Term Stability for Clinical Translation of RNAi

(2019) Nadia R. Ayat et al.   Nucleic Acid Therapeutics

Ocular gene therapies in clinical practice: viral vectors and nonviral alternatives

(2019) Thierry Bordet et al.   DRUG DISCOVERY TODAY

Gene delivery to the rat retina by non-viral vectors based on chloroquine-containing cationic niosomes

(2019) Mohamed Mashal et al.   JOURNAL OF CONTROLLED RELEASE

Non-viral Gene Therapy for Stargardt Disease with ECO/pRHO-ABCA4 Self-Assembled Nanoparticles

(2019) Da Sun et al.   MOLECULAR THERAPY

Synthesis and Evaluation of pH-Sensitive Multifunctional Lipids for Efficient Delivery of CRISPR/Cas9 in Gene Editing

(2018) Da Sun et al.   BIOCONJUGATE CHEMISTRY

Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye

(2018) Gerard A. Rodrigues et al.   PHARMACEUTICAL RESEARCH

Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations

(2017) William A. Beltran et al.   MOLECULAR THERAPY

Revisiting the role of sucrose in PLGA-PEG nanocarrier for potential intranasal delivery

(2017) A. Bonaccorso et al.   PHARMACEUTICAL DEVELOPMENT AND TECHNOLOGY

Targeted Multifunctional Lipid ECO Plasmid DNA Nanoparticles as Efficient Non-viral Gene Therapy for Leber’s Congenital Amaurosis

(2017) Da Sun et al.   Molecular Therapy-Nucleic Acids

Nanoparticle-based drug delivery systems: a commercial and regulatory outlook as the field matures

(2016) Héloïse Ragelle et al.   Expert Opinion on Drug Delivery

Cone-Specific Promoters for Gene Therapy of Achromatopsia and Other Retinal Diseases

(2016) Guo-Jie Ye et al.   HUMAN GENE THERAPY

Adeno-associated virus (AAV) vectors in cancer gene therapy

(2016) Jorge L. Santiago-Ortiz et al.   JOURNAL OF CONTROLLED RELEASE

Facts and evidences on the lyophilization of polymeric nanoparticles for drug delivery

(2016) Pedro Fonte et al.   JOURNAL OF CONTROLLED RELEASE

Manufacturing of recombinant adeno-associated viral vectors for clinical trials

(2016) Nathalie Clément et al.   Molecular Therapy-Methods & Clinical Development

Multifunctional pH-Sensitive Amino Lipids for siRNA Delivery

(2015) Maneesh Gujrati et al.   BIOCONJUGATE CHEMISTRY

Non-viral therapeutic approaches to ocular diseases: An overview and future directions

(2015) Rahel Zulliger et al.   JOURNAL OF CONTROLLED RELEASE

Delivery strategies for treatment of age-related ocular diseases: From a biological understanding to biomaterial solutions

(2015) Vianney Delplace et al.   JOURNAL OF CONTROLLED RELEASE

Self-Assembly of a Multifunctional Lipid With Core-Shell Dendrimer DNA Nanoparticles Enhanced Efficient Gene Delivery at Low Charge Ratios into RPE Cells

(2015) Da Sun et al.   MACROMOLECULAR BIOSCIENCE

Investor Outlook: Significance of the Positive LCA2 Gene Therapy Phase III Results

(2015) Joshua Schimmer et al.   Human Gene Therapy Clinical Development

Multifunctional Cationic Lipid-Based Nanoparticles Facilitate Endosomal Escape and Reduction-Triggered Cytosolic siRNA Release

(2014) Maneesh Gujrati et al.   MOLECULAR PHARMACEUTICS

Persistence of non-viral vector mediated RPE65 expression: Case for viability as a gene transfer therapy for RPE-based diseases

(2013) Adarsha Koirala et al.   JOURNAL OF CONTROLLED RELEASE

Design and evaluation of new pH-sensitive amphiphilic cationic lipids for siRNA delivery

(2013) Anthony S. Malamas et al.   JOURNAL OF CONTROLLED RELEASE

Efficacy of Codelivery of Dual AAV2/5 Vectors in the Murine Retina and Hippocampus

(2012) Arpad Palfi et al.   HUMAN GENE THERAPY

Clinical applications of retinal gene therapy

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

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

(2012) J. Bennett et al.   Science Translational Medicine

Immune Responses to AAV in Clinical Trials

(2011) Federico Mingozzi et al.   CURRENT GENE THERAPY

Focus on Molecules: ABCA4 (ABCR) – An import-directed photoreceptor retinoid flipase

(2009) Jack M. Sullivan   EXPERIMENTAL EYE RESEARCH

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

(2009) Francesca Simonelli et al.   MOLECULAR THERAPY

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

Freeze drying of human serum albumin (HSA) nanoparticles with different excipients

(2008) Marion G. Anhorn et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

Usher syndrome: Animal models, retinal function of Usher proteins, and prospects for gene therapy

(2007) David S. Williams   VISION RESEARCH