Immunogenicity assessment of AAV-based gene therapies: an IQ consortium industry white paper

T Yang, M Braun, W Lembke, F McBlane… - … Therapy Methods & …, 2022 - cell.com
Immunogenicity has imposed a challenge to efficacy and safety evaluation of adeno-
associated virus (AAV) vector-based gene therapies. Mild to severe adverse events …

Subretinal therapy: technological solutions to surgical and immunological challenges

R Ladha, LE Caspers, F Willermain… - Frontiers in medicine, 2022 - frontiersin.org
Recent advances in ocular gene and cellular therapy rely on precisely controlled subretinal
delivery. Due to its inherent limitations, manual delivery can lead to iatrogenic damage to the …

Advances in ophthalmic optogenetics: approaches and applications

PP Prosseda, M Tran, T Kowal, B Wang, Y Sun - Biomolecules, 2022 - mdpi.com
Recent advances in optogenetics hold promise for vision restoration in degenerative eye
diseases. Optogenetics refers to techniques that use light to control the cellular activity of …

The AAV2. 7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2

M Cui, Q Su, M Yip, J McGowan, C Punzo, G Gao… - Gene therapy, 2024 - nature.com
Recombinant adeno-associated virus (rAAV) vectors are currently the only proven vehicles
for treating ophthalmological diseases through gene therapy. A wide range of gene therapy …

Gene therapy for age-related macular degeneration: Potential, feasibility, and pitfalls

ST Berkowitz, AP Finn - Current Opinion in Ophthalmology, 2024 - journals.lww.com
The present review discusses emerging gene therapy options which could foster new, more
durable nAMD and nnAMD therapeutics. These options will need refinement with regards to …

[HTML][HTML] Therapeutic applications of adeno-associated virus (AAV) gene transfer of HLA-G in the eye

BC Gilger, ML Hirsch - International Journal of Molecular Sciences, 2022 - mdpi.com
The purpose of this paper is to review human leukocyte antigen G (HLA-G) in the eye, its
role in immune tolerance, and the potential therapeutic use of AAV gene transfer and …

From bench to bed: the current genome editing therapies for glaucoma

M He, R Rong, D Ji, X **a - Frontiers in Cell and Developmental …, 2022 - frontiersin.org
Glaucoma is a group of optic neuropathies featured by degeneration of retinal ganglion cells
and loss of their axons in the optic nerve. The only currently approved therapies focus on …

Inhibition of experimental autoimmune uveitis by intravitreal AAV-Equine-IL10 gene therapy

E Crabtree, K Uribe, SM Smith, D Roberts, JH Salmon… - PLoS …, 2022 - journals.plos.org
Equine recurrent uveitis (ERU) is a spontaneous, painful, and vision threatening disease
affecting up to 25% of equine populations worldwide. Current treatments of ERU are non …

[HTML][HTML] Immunogenicity of novel AAV capsids for retinal gene therapy

M Gehrke, M Diedrichs-Möhring, J Bogedein, H Büning… - Cells, 2022 - mdpi.com
Objectives: AAV vectors are widely used in gene therapy, but the prevalence of neutralizing
antibodies raised against AAV serotypes in the course of a natural infection, as well as …

Immunology of Retinitis Pigmentosa and Gene Therapy–Associated Uveitis

P Yang, D Mustafi, KL Pepple - Cold Spring Harbor …, 2024 - pmc.ncbi.nlm.nih.gov
The underlying immune state of inherited retinal degenerations (IRDs) and retinitis
pigmentosa (RP) has been an emerging area of interest, where in the consequences have …