[HTML][HTML] Adeno-Associated Virus Therapies: Pioneering Solutions for Human Genetic Diseases

D Liu, T Li, L Liu, X Che, X Li, C Liu, G Wu - Cytokine & Growth Factor …, 2024 - Elsevier
Adeno-associated virus (AAV) has emerged as a fundamental component in the gene
therapy landscape, widely acknowledged for its effectiveness in therapeutic gene delivery …

Recent Advancements and Applications of Ophthalmic Gene Therapy Strategies: A Breakthrough in Ocular Therapeutics

R Maurya, A Vikal, RK Narang, P Patel… - Experimental Eye …, 2024 - Elsevier
Over the past twenty years, ocular gene therapy has primarily focused on addressing
diseases linked to various genetic factors. The eye is an ideal candidate for gene therapy …

Comparison of SNCG and NEFH promoter–driven expression of human SIRT1 expression in a mouse model of glaucoma

N O'Neill, M Meng, B Chaqour, K Dine… - … Vision Science & …, 2024 - tvst.arvojournals.org
Purpose: Adeno-associated virus (AAV) demonstrates promise in delivering therapeutic
genes to retinal ganglion cells (RGCs). Delivery of neuroprotective genes is constrained by …

[HTML][HTML] Berries and Their Active Compounds in Prevention of Age-Related Macular Degeneration

X Li, L Zhao, B Zhang, S Wang - Antioxidants, 2024 - mdpi.com
Age-related macular degeneration (AMD) is a leading cause of vision loss in the elderly,
significantly diminishing quality of life. Currently, there is no available treatment to reverse …

[HTML][HTML] Exercise-Induced Reduction of IGF1R Sumoylation Attenuates Neuroinflammation in APP/PS1 Transgenic Mice

Y Chen, X Chen, Z Luo, X Kang, Y Ge, R Wan… - Journal of Advanced …, 2024 - Elsevier
Abstract Introduction Alzheimer's Disease (AD), a progressive neurodegenerative disorder,
is marked by cognitive deterioration and heightened neuroinflammation. The influence of …

Neuropathological Findings in Nonclinical Species Following Administration of Adeno-Associated Virus (AAV)-Based Gene Therapy Vectors

B Bolon, E Buza, E Galbreath, J Wicks… - Toxicologic …, 2024 - journals.sagepub.com
Adeno-associated virus (AAV) gene therapy vectors are an accepted platform for treating
severe neurological diseases. Test article (TA)-related and procedure-related …

[HTML][HTML] Ocular Gene Therapy: An Overview of Viral Vectors, Immune Responses, and Future Directions

L Banou, S Sarrafpour, CC Teng… - The Yale Journal of …, 2024 - pmc.ncbi.nlm.nih.gov
Ocular gene therapy has rapidly advanced from proof-of-concept studies to clinical trials by
exploiting the unique advantages of the eye, including its easy accessibility, relative immune …

[HTML][HTML] Infantile Nystagmus Syndrome—Associated Inherited Retinal Diseases: Perspectives from Gene Therapy Clinical Trials

X Gong, RW Hertle - Life, 2024 - mdpi.com
Inherited retinal diseases (IRDs) are a clinically and genetically diverse group of progressive
degenerative disorders that can result in severe visual impairment or complete blindness …

Therapeutic Potential of AAV2-shmTOR Gene Therapy in Reducing Retinal Inflammation and Preserving Endothelial Integrity in Age-Related Macular Degeneration

J Kim, SY Moon, HG Kang, HJ Kim, JS Choi, SHS Lee… - 2024 - researchsquare.com
Age-related macular degeneration (AMD) is a prevalent retinal disorder that leads to central
vision loss, mainly due to chronic inflammation. Tumor necrosis factor-alpha (TNF-α) is a …

Ocular RNA nanomedicine: engineered delivery nanoplatforms in treating eye diseases

Y Zhang, Y Shi, MM Khan, F Xiao, W Chen, W Tao… - Trends in …, 2024 - cell.com
Ocular disorders remain a major global health challenge with unmet medical needs. RNA
nanomedicine has shown significant therapeutic benefits and safety profiles in patients with …