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

2019 ◽  
Vol 27 (1) ◽  
pp. 118-129 ◽  
Author(s):  
Ruslan Grishanin ◽  
Brian Vuillemenot ◽  
Pallavi Sharma ◽  
Annahita Keravala ◽  
Judith Greengard ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Macular Degeneration ◽  
Age Related Macular Degeneration ◽  
Therapy Approach ◽  
Preclinical Evaluation ◽  
Gene Therapy Approach ◽  
Novel Gene ◽  
Age Related

scholarly journals Non-medical treatment for late age-related macular degeneration

2021 ◽  
Vol 21 (4) ◽  
pp. 215-219
Author(s):  
A.K. Drakon ◽  
◽  
A.G. Kurguzova ◽  
V.M. Sheludchenko ◽  
N.B. Korchazhkina ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Medical Treatment ◽  
Macular Degeneration ◽  
Retinal Pigment ◽  
Embryonic Stem ◽  
Age Related Macular Degeneration ◽  
Target Cells ◽  
Specific Gene ◽  
Age Related

Age-related macular degeneration (AMD) is the leading cause of blindness in people over 55 in developed countries. Moreover, the number of these patients will increase growth as life expectancy increases. It is estimated that late AMD accounts for half of blindness and low vision cases in European countries. A myriad of studies is currently underway to discover cutting-edge, effective therapeutic modalities. Gene therapy is a novel alternative to regular intravitreal injections of anti-VEGF agents for late wet AMD. This technique’s heart is a specific gene delivery to target cells to generate natural VEGF inhibitors. Gene therapy affecting the complement system to deactivate its end product, the membrane attack complex, is reasonable in late atrophic AMD. Studies on stem cell therapy for late atrophic AMD undergo as well. It was demonstrated that retinal pigment epithelium (RPE) cells derived from human embryonic stem cells or induced pluripotent stem cells express typical RPE markers that can phagocytize photoreceptor segments. Electrical stimulation and magnet therapy are already introduced into clinical practice to rehabilitate patients with late AMD. Magnetic and electrical fields improve impulse transmitting, activate intracellular and tissue regeneration of the retina. Recent findings are promising but require further in-depth studies. Keywords: age-related macular degeneration, retinal scar, gene therapy, stem cells, physiotherapy, rehabilitative medicine. For citation: Drakon A.K., Kurguzova A.G., Sheludchenko V.M., Korchazhkina N.B. Non-medical treatment for late age-related macular degeneration. Russian Journal of Clinical Ophthalmology. 2021;21(4):215–219 (in Russ.). DOI: 10.32364/2311-7729-2021-21-4-215-219.

scholarly journals The role of hypoxia-inducible factors in neovascular age-related macular degeneration: a gene therapy perspective

2019 ◽  
Vol 77 (5) ◽  
pp. 819-833 ◽  
Author(s):  
Parviz Mammadzada ◽  
Pablo M. Corredoira ◽  
Helder André
Keyword(s):  
Gene Therapy ◽  
Macular Degeneration ◽  
Clinical Phenotype ◽  
Therapeutic Strategies ◽  
Age Related Macular Degeneration ◽  
Hypoxia Inducible Factors ◽  
Ocular Angiogenesis ◽  
Gene Therapies ◽  
Age Related

AbstractUnderstanding the mechanisms that underlie age-related macular degeneration (AMD) has led to the identification of key molecules. Hypoxia-inducible transcription factors (HIFs) have been associated with choroidal neovascularization and the progression of AMD into the neovascular clinical phenotype (nAMD). HIFs regulate the expression of multiple growth factors and cytokines involved in angiogenesis and inflammation, hallmarks of nAMD. This knowledge has propelled the development of a new group of therapeutic strategies focused on gene therapy. The present review provides an update on current gene therapies in ocular angiogenesis, particularly nAMD, from both basic and clinical perspectives.

scholarly journals Gene therapy for neovascular age-related macular degeneration: rationale, clinical trials and future directions

2020 ◽  
pp. bjophthalmol-2020-316195 ◽  
Author(s):  
Thales Antonio Cabral de Guimaraes ◽  
Michalis Georgiou ◽  
James W B Bainbridge ◽  
Michel Michaelides
Keyword(s):  
Gene Therapy ◽  
Clinical Trials ◽  
Macular Degeneration ◽  
Age Related Macular Degeneration ◽  
Current Status ◽  
Routes Of Administration ◽  
Age Related ◽  
Potential Safety ◽  
Early Phase Clinical Trials ◽  
Endothelial Growth Factor

Age-related macular degeneration (AMD) is one of the leading causes of irreversible blindness in the developed world. Antivascular endothelial growth factor therapy has transformed the management and outcome of neovascular AMD (nAMD), although the need for repeated intravitreal injections—even lifelong—and the related complications, high drug costs, frequent clinic visits and repeated imaging have resulted in an enormous burden both to healthcare systems and patients. The application of gene therapy approaches for sustained delivery of a range of antiangiogenic proteins has the promise of helping to address these aforementioned challenges. A number of early phase clinical trials of gene therapy in nAMD have provided encouraging results, with many more ongoing or anticipated. There remain significant areas of controversy, including regarding the optimal treatment targets, routes of administration and potential safety concerns. In this review we aim to provide an update of the current status of gene therapy for nAMD and briefly discuss future prospects.

Bax-Induced Apoptosis as a Novel Gene Therapy Approach for Carcinoma of the Cervix

2001 ◽  
Vol 83 (2) ◽  
pp. 370-377 ◽  
Author(s):  
Warner K. Huh ◽  
Jesus Gomez-Navarro ◽  
Waleed O. Arafat ◽  
Jialing Xiang ◽  
Parameshwar J. Mahasreshti ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Therapy Approach ◽  
Gene Therapy Approach ◽  
Novel Gene ◽  
Induced Apoptosis

scholarly journals Nanotechnology for Age-Related Macular Degeneration

Pharmaceutics ◽  
2021 ◽  
Vol 13 (12) ◽  
pp. 2035
Author(s):  
Bo Yang ◽  
Ge Li ◽  
Jiaxin Liu ◽  
Xiangyu Li ◽  
Shixin Zhang ◽  
...  
Keyword(s):  
Drug Delivery ◽  
Gene Therapy ◽  
Macular Degeneration ◽  
Vision Loss ◽  
Cumulative Risk ◽  
Age Related Macular Degeneration ◽  
Delivery Methods ◽  
Age Related ◽  
Therapy Strategies ◽  
Endothelial Growth Factor

Age-related macular degeneration (AMD) is a degenerative eye disease that is the leading cause of irreversible vision loss in people 50 years and older. Today, the most common treatment for AMD involves repeated intravitreal injections of anti-vascular endothelial growth factor (VEGF) drugs. However, the existing expensive therapies not only cannot cure this disease, they also produce a variety of side effects. For example, the number of injections increases the cumulative risk of endophthalmitis and other complications. Today, a single intravitreal injection of gene therapy products can greatly reduce the burden of treatment and improve visual effects. In addition, the latest innovations in nanotherapy provide the best drug delivery alternative for the treatment of AMD. In this review, we discuss the development of nano-drug delivery systems and gene therapy strategies for AMD in recent years. In addition, we discuss some novel targeting strategies and the potential application of these delivery methods in the treatment of AMD. Finally, we also propose that the combination of CRISPR/Cas9 technology with a new non-viral delivery system may be promising as a therapeutic strategy for the treatment of AMD.

Three-Year Follow-Up of Phase 1 and 2a rAAV.sFLT-1 Subretinal Gene Therapy Trials for Exudative Age-Related Macular Degeneration

2019 ◽  
Vol 204 ◽  
pp. 113-123 ◽  
Author(s):  
Elizabeth P. Rakoczy ◽  
Aaron L. Magno ◽  
Chooi-May Lai ◽  
Cora M. Pierce ◽  
Mariapia A. Degli-Esposti ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Macular Degeneration ◽  
Phase 1 ◽  
Age Related Macular Degeneration ◽  
Age Related

Gene Therapy, Stem Cell Treatment and Retinal Implant in Dry-Form (Non-Neovascular) Age-Related Macular Degeneration

2017 ◽  
pp. 149-154
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Macular Degeneration ◽  
Age Related Macular Degeneration ◽  
Retinal Implant ◽  
Age Related ◽  
Functional Vision ◽  
Stem Cell Treatment ◽  
End Stage ◽  
Legal Blindness

Nowadays there is no treatment option except antioxidant support which decelerates the progression of dry-type age-related macular degeneration (ARMD) which is one of the most common reasons for legal blindness in elderly people. Promising outcomes have been obtained with stem cell treatment and gene therapy in dry-type ARMD in the recent studies which are done in the light of the advanced bioengineering developments. In addition, very rapid advances have been developing related to the retinal prosthesis implantation which is performed to regain their functional vision in end-stage ARMD patients.

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