scispace - formally typeset
R

Rajeshwari D. Koilkonda

Researcher at Bascom Palmer Eye Institute

Publications -  21
Citations -  989

Rajeshwari D. Koilkonda is an academic researcher from Bascom Palmer Eye Institute. The author has contributed to research in topics: Retinal ganglion & Mitochondrial DNA. The author has an hindex of 12, co-authored 21 publications receiving 808 citations. Previous affiliations of Rajeshwari D. Koilkonda include University of Miami.

Papers
More filters
Journal ArticleDOI

Gene Therapy for Leber Hereditary Optic Neuropathy: Initial Results.

TL;DR: No serious safety problems were observed in the first 5 participants enrolled in this phase I trial of virus-based gene transfer in this mitochondrial disorder and additional study follow-up of these and additional participants planned for the next 4 years is needed to confirm these preliminary observations.
Journal ArticleDOI

Gene Therapy for Leber Hereditary Optic Neuropathy: Low- and Medium-Dose Visual Results

TL;DR: Allotopic gene therapy for LHON at low and medium doses seems to be safe and does not damage the temporal retinal nerve fiber layer, opening the door next for testing of the high dose.
Journal ArticleDOI

Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber’s hereditary optic neuropathy in a mouse model

TL;DR: The adenoassociated virus cassette accommodates genes of up to ∼5 kb in length, thus providing a platform for introduction of almost any mitochondrial gene and perhaps even allowing insertion of DNA encompassing large deletions of mtDNA into the organelle of adults.
Journal ArticleDOI

Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system.

TL;DR: Although it had approximately two-thirds the efficiency of GFP, the expression of normal human ND4 in murine mitochondria did not induce the loss of RGCs, ATP synthesis, or PERG amplitude, suggesting that allotopic ND4 may be safe for the treatment of patients with Leber hereditary optic neuropathy.
Journal ArticleDOI

Safety and Effects of the Vector for the Leber Hereditary Optic Neuropathy Gene Therapy Clinical Trial

TL;DR: expression of the authors' allotopic ND4 vector in the ex vivo human eye, safety of the test article, rescue of the LHON mouse model, and the severe irreversible loss of visual function in LHON support clinical testing with mutated G11778A mitochondrial DNA in patients.