Progress in Gene Therapy to Prevent Retinal Ganglion Cell Loss in Glaucoma and Leber’s Hereditary Optic Neuropathy

Triple Vectors Expand AAV Transfer Capacity in the Retina

(2018) Andrea Maddalena et al.   MOLECULAR THERAPY

Safety of rAAV2/2- ND4 Gene Therapy for Leber Hereditary Optic Neuropathy

(2018) Catherine Vignal et al.   OPHTHALMOLOGY

Comparing successful gene knock-in efficiencies of CRISPR/Cas9 with ZFNs and TALENs gene editing systems in bovine and dairy goat fetal fibroblasts

(2018) Hui LIU et al.   Journal of Integrative Agriculture

Glaucoma

(2017) Jost B Jonas et al.   LANCET

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

Unexpected mutations after CRISPR–Cas9 editing in vivo

(2017) Kellie A Schaefer et al.   NATURE METHODS

Gene Therapy for Leber Hereditary Optic Neuropathy

(2017) John Guy et al.   OPHTHALMOLOGY

CRISPR-Cas9–based treatment of myocilin-associated glaucoma

(2017) Ankur Jain et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

In vivo genome editing as a potential treatment strategy for inherited retinal dystrophies

(2017) Mert Yanik et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Vitamin B 3 modulates mitochondrial vulnerability and prevents glaucoma in aged mice

(2017) Pete A. Williams et al.   SCIENCE

Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice

(2017) Wenhan Yu et al.   Nature Communications

CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors

(2017) Rasmus O. Bak et al.   Cell Reports

A Novel Retinal Ganglion Cell Promoter for Utility in AAV Vectors

(2017) Killian S. Hanlon et al.   Frontiers in Neuroscience

Spatially and Temporally Regulated NRF2 Gene Therapy Using Mcp-1 Promoter in Retinal Ganglion Cell Injury

(2017) Kosuke Fujita et al.   Molecular Therapy-Methods & Clinical Development

Genetic manipulation for inherited neurodegenerative diseases: myth or reality?

(2016) Patrick Yu-Wai-Man   BRITISH JOURNAL OF OPHTHALMOLOGY

Differential effects of BDNF and neurotrophin 4 (NT4) on endocytic sorting of TrkB receptors

(2016) Catia C. Proenca et al.   JOURNAL OF NEUROCHEMISTRY

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

In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

(2016) Keiichiro Suzuki et al.   NATURE

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Gene Therapy for Leber Hereditary Optic Neuropathy

(2016) William J. Feuer et al.   OPHTHALMOLOGY

Correction: Blindness and Glaucoma: A Multicenter Data Review from 7 Academic Eye Clinics

(2016) Luca Rossetti et al.   PLoS One

Vitrectomy Before Intravitreal Injection of AAV2/2 Vector Promotes Efficient Transduction of Retinal Ganglion Cells in Dogs and Nonhuman Primates

(2016) Kizito-Tshitoko Tshilenge et al.   Human Gene Therapy Methods

Ocular Immune Privilege and Transplantation

(2016) Andrew W. Taylor   Frontiers in Immunology

Significant changes in endogenous retinal gene expression assessed 1 year after a single intraocular injection of AAV-CNTF or AAV-BDNF

(2016) Chrisna J LeVaillant et al.   Molecular Therapy-Methods & Clinical Development

Efficacy and Safety of rAAV2-ND4 Treatment for Leber’s Hereditary Optic Neuropathy

(2016) Xing Wan et al.   Scientific Reports

Selective Elimination of Mitochondrial Mutations in the Germline by Genome Editing

(2015) Pradeep Reddy et al.   CELL

Is there treatment for Leber hereditary optic neuropathy?

(2015) Jason H. Peragallo et al.   CURRENT OPINION IN OPHTHALMOLOGY

Retinal gene delivery by adeno-associated virus (AAV) vectors: Strategies and applications

(2015) Christian Schön et al.   EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS

What Is Next for Retinal Gene Therapy?

(2015) Luk H. Vandenberghe   Cold Spring Harbor Perspectives in Medicine

Efficient Mitochondrial Genome Editing by CRISPR/Cas9

(2015) Areum Jo et al.   Biomed Research International

Nuclear expression of mitochondrial ND4 leads to the protein assembling in complex I and prevents optic atrophy and visual loss

(2015) Hélène Cwerman-Thibault et al.   Molecular Therapy-Methods & Clinical Development

BDNF impairment is associated with age-related changes in the inner retina and exacerbates experimental glaucoma

(2014) Vivek Gupta et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9

(2014) Rajat M. Gupta et al.   JOURNAL OF CLINICAL INVESTIGATION

CRISPR-Cas systems for editing, regulating and targeting genomes

(2014) Jeffry D Sander et al.   NATURE BIOTECHNOLOGY

Global Prevalence of Glaucoma and Projections of Glaucoma Burden through 2040

(2014) Yih-Chung Tham et al.   OPHTHALMOLOGY

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

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

(2013) Ivana Trapani et al.   EMBO Molecular Medicine

Why does Brain Metabolism not Favor Burning of Fatty Acids to Provide Energy? - Reflections on Disadvantages of the Use of Free Fatty Acids as Fuel for Brain

(2013) Peter Schönfeld et al.   JOURNAL OF CEREBRAL BLOOD FLOW AND METABOLISM

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Rates of visual field progression in clinical glaucoma care

(2012) Anders Heijl et al.   ACTA OPHTHALMOLOGICA

Long-Term Gene Therapy Causes Transgene-Specific Changes in the Morphology of Regenerating Retinal Ganglion Cells

(2012) Jennifer Rodger et al.   PLoS One

Good news–bad news: the Yin and Yang of immune privilege in the eye

(2012) John V. Forrester et al.   Frontiers in Immunology

Gene therapy for retinal ganglion cell neuroprotection in glaucoma

(2011) A M Wilson et al.   GENE THERAPY

Gene supplementation therapy for recessive forms of inherited retinal dystrophies

(2011) A J Smith et al.   GENE THERAPY

Glaucoma

(2011) Harry A Quigley   LANCET

The molecular basis of retinal ganglion cell death in glaucoma

(2011) Mohammadali Almasieh et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Induction of Rapid and Highly Efficient Expression of the Human ND4 Complex I Subunit in the Mouse Visual System by Self-complementary Adeno-Associated Virus

(2010) Rajeshwari D. Koilkonda   ARCHIVES OF OPHTHALMOLOGY

Neuroprotection in glaucoma – Is there a future role?

(2010) Abeir Baltmr et al.   EXPERIMENTAL EYE RESEARCH

Does elevated intraocular pressure reduce retinal TRKB-mediated survival signaling in experimental glaucoma?

(2009) Ying Guo et al.   EXPERIMENTAL EYE RESEARCH

Optimized Allotopic Expression of the Human Mitochondrial ND4 Prevents Blindness in a Rat Model of Mitochondrial Dysfunction

(2008) Sami Ellouze et al.   AMERICAN JOURNAL OF HUMAN GENETICS

BAG1 promotes axonal outgrowth and regeneration in vivo via Raf-1 and reduction of ROCK activity

(2008) Véronique Planchamp et al.   BRAIN

Cellular tropism and transduction properties of seven adeno-associated viral vector serotypes in adult retina after intravitreal injection

(2008) M Hellström et al.   GENE THERAPY

Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: Effects of serotype and site of administration

(2007) Ji-jing Pang et al.   VISION RESEARCH