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Gene therapy

Dynamic BMP gene expression regulation in chick RPE during recovery from short term optical defocus and form-deprivation

Yan Zhang, Qiurong Zhu, [ ... ], Christine F. Wildsoet

In utero adeno-associated virus (AAV)-mediated gene delivery targeting sensory and supporting cells in the embryonic mouse inner ear

Carla Maria Barbosa Spinola, Jacques Boutet de Monvel, [ ... ], Raphaël Etournay

Clinician’s perspectives on gene therapy for Alzheimer’s disease: A qualitative study

Lilly Kelemen, Ishika Gupta, [ ... ], Nathan A. Boucher

Clinical and analytical validation of an 82-gene comprehensive genome-profiling panel for identifying and interpreting variants responsible for inherited retinal dystrophies

Jacqueline Chan, Jolyon Holdstock, [ ... ], Akiko Maeda

Electric pulse exposure reduces AAV8 dosage required to transduce HepG2 cells

Yizhou Yao, Robert W. Holdcraft, Susan C. Hagness, John H. Booske

A phase I clinical trial of oncolytic adenovirus mediated suicide and interleukin-12 gene therapy in patients with recurrent localized prostate adenocarcinoma

Shyam Nyati, Hans Stricker, [ ... ], Farzan Siddiqui

AAV2/4-RS1 gene therapy in the retinoschisin knockout mouse model of X-linked retinoschisis

Brittni A. Scruggs, Sajag Bhattarai, [ ... ], Arlene V. Drack

Multiple criteria decision analysis for therapeutic innovations in a hemophilia care center: A pilot study of the organizational impact of innovation in hemophilia care management

Karen Beny, Amélie Dubromel, [ ... ], Claude Dussart

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

Elizabeth Crabtree, Katy Uribe, [ ... ], Brian C. Gilger

mTOR inhibition as a novel gene therapeutic strategy for diabetic retinopathy

Steven Hyun Seung Lee, Joo Yong Lee, [ ... ], Heuiran Lee

Assessment of the percentage of full recombinant adeno-associated virus particles in a gene therapy drug using CryoTEM

Mathieu Colomb-Delsuc, Roman Raim, [ ... ], Ida-Maria Sintorn

Improved targeting of human CD4⁺ T cells by nanobody-modified AAV2 gene therapy vectors

Martin V. Hamann, Niklas Beschorner, [ ... ], Joachim Hauber

Low efficacy of recombinant SV40 in Ugt1a1^-/- mice with severe inherited hyperbilirubinemia

Xiaoxia Shi, Giulia Bortolussi, [ ... ], Piter J. Bosma

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