Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors

CN Kay, RC Ryals, GV Aslanidi, SH Min, Q Ruan… - PloS one, 2013 - journals.plos.org
novel AAV vectors optimized for photoreceptor transduction following intravitreal delivery
Transduction efficiencies of self-complimentary, capsid-mutant and unmodified AAV vectors

Improvement of photoreceptor targeting via intravitreal delivery in mouse and human retina using combinatory rAAV2 capsid mutant vectors

CA Reid, KJ Ertel, DM Lipinski - Investigative ophthalmology & …, 2017 - iovs.arvojournals.org
… Seventy-two hours post transfection, purification of AAV vectorusing a minimally invasive
intravitreal delivery technique, wherein a small volume of undiluted capsid mutant virus vector

Engineered capsids for efficient gene delivery to the retina and cornea

A Frederick, J Sullivan, L Liu, M Adamowicz… - Human Gene …, 2020 - liebertpub.com
… Notably, both intravitreal and subretinal delivery of AAV vectors … capsids for specific targeting
of AAV vectors. The interaction … in photoreceptors from an AAV5 and an AAV2-HBKO vector, …

Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach

FM Mowat, KR Gornik, A Dinculescu, SL Boye… - Gene therapy, 2014 - nature.com
intravitreal injection of vector and more efficient targeting of inner retinal cells to introduce
novel … An alternative approach is to target the photoreceptors and RPE via intravitreal injection. …

Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs

RF Boyd, DG Sledge, SL Boye, SE Boye… - Gene therapy, 2016 - nature.com
… of novel AAV vectors to transduce photoreceptors in dogs following IVT. We found both IRBP
Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors. …

Evaluation of photoreceptor transduction efficacy of capsid-modified adeno-associated viral vectors following intravitreal and subretinal delivery in sheep

M Ross, A Obolensky, E Averbukh, R Ezra-Elia… - Human Gene …, 2020 - liebertpub.com
… Consequently, when treatment is targeted to cone photoreceptors, the vector should be
injected in the central and most sensitive part of the retina, namely macular/foveal area. Third, …

Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness

ML Scalabrino, SL Boye, KMH Fransen… - Human molecular …, 2015 - academic.oup.com
… epithelium and photoreceptors are efficiently targeted by various AAV serotypes following
delivery to the subretinal space. RGCs are efficiently transduced following vitreal delivery (38,…

AAV for gene therapy in ocular diseases: progress and prospects

X He, Y Fu, L Ma, Y Yao, S Ge, Z Yang, X Fan - Research, 2023 - spj.science.org
Intraocular injection of purified recombination ciliary … applied in clinical trials to target
photoreceptors (rods and cones) [… shows that those 2 novel AAV variants can efficiently target

Developing new vectors for retinal gene therapy

EA Zin, BE Ozturk, D Dalkara… - Cold Spring …, 2023 - perspectivesinmedicine.cshlp.org
… to AAV development in the context of intricate intraocular spaces… for photoreceptors compared
to AAVs. For these reasons, … yield a toolbox of AAV vectors capable of targeting each of the …

Intravitreal injection of a rationally designed AAV capsid library in non-human primate identifies variants with enhanced retinal transduction and neutralizing antibody …

PC Kellish, D Marsic, SM Crosson, S Choudhury… - Molecular Therapy, 2023 - cell.com
… While delivering AAV in situ to photoreceptors and RPE via subretinal injection leads to efficient
transduction of these cells, it is not a suitable route for targeting … of our novel AAV capsid …