Abstract
Deadline for Manuscript Submission: November 15, 2024
The first recombinant adeno-associated virus (AAV) vector licensed by the FDA for human use was Luxturna (voretigene neparvovec-rzl) in 2017, a vector designed to treat inherited eye disease, inherited retinal dystrophy due to biallelic RPE65 mutation. Numerous other gene therapies for eye disease, both inherited and acquired have been pursued during the years that followed. Such applications are attractive because the small size of ocular tissues, such as the retina, and its compartmentalized nature enable small doses of vector to have clinically significant effects. Furthermore, the eye has been considered as relatively immune privileged, potentially improving the safety profile and requiring little or no immune suppression to achieve vector persistence. However, as numerous potential applications of gene therapy have progressed in clinical development, some of the prior assumptions have been challenged and other weaknesses and opportunities have been demonstrated. Human Gene Therapy is planning a special issue devoted to Gene Therapy for Diseases of the Eye early in 2025. We are seeking original papers, review articles, and minireviews addressing in ocular gene therapy. Such papers may include, but are not limited to: Studies of novel AAV, lentiviral and nonviral gene therapy vectors for ocular gene therapy Proof-of-concept studies demonstrating bioefficacy of gene therapies for new disease targets within ophthalmology Clinical development studies with ocular gene therapies, including IND-enabling pharmacology and toxicology studies Methods and protocols, including new surgical protocols, facilitating gene therapy for eye disease
The target deadline for submissions is November 15, 2024. Submissions after that date may not be reviewed in time for the special issue.
https://mc.manuscriptcentral.com/hum
Special Issue: Ocular Gene Therapy