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Defining Phenotype, Tropism, and Retinal Gene Therapy Using Adeno-Associated Viral Vectors (AAVs) in New-Born Brown Norway Rats with a Spontaneous Mutation in

• Authors : Nanda, Boon; C Henrique, Alves; Aat A, Mulder

Subjects: Ependymoglial Cells; Genetic Vectors; Nerve Tissue Proteins

• Source: International Journal of Molecular Sciences

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Defining Phenotype, Tropism, and Retinal Gene Therapy Using Adeno-Associated Viral Vectors (AAVs) in New-Born Brown Norway Rats with a Spontaneous Mutation in Crb1

• Authors : Aat A. Mulder; Carolina R. Jost; Abraham J. Koster ...

Subjects: 0301 basic medicine; Mutant; 030105 genetics & heredity

• Source: International Journal of Molecular Sciences, 22(7):3563. Multidisciplinary Digital Publishing Institute (MDPI)International Journal of Molecular Sciences, 22(7). MDPIInternational Journal

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The Signalling Role of the αvβ5-Integrin Can Impact the Efficacy of AAV in Retinal Gene Therapy

• Authors : Therese Cronin; Emeline F. Nandrot; Jean Bennett ...

Subjects: Pathology; medicine.medical_specialty; [SDV.BIO]Life Sciences [q-bio]/Biotechnology

• Source: PharmaceuticalsPharmaceuticals, MDPI, 2012, 5 (5), pp.447-459. ⟨10.3390/ph5050447⟩Pharmaceuticals; Volume 5; Issue 5; Pages: 447-459Pharmaceuticals, Vol 5, Iss 5, Pp 447-459

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Hypotrichosis and juvenile macular dystrophy caused by CDH3 mutation: A candidate disease for retinal gene therapy

• Authors : Richard Holt; Stephanie Halford; Mandeep S. Singh

Subjects: Male; 0301 basic medicine; Pathology

• Source: Scientific Reports

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Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial

• Authors : MacLaren, RE; Groppe, M; Barnard, AR

Subjects: Adult; Male; medicine.medical_specialty

• Source: The Lancet (London), 383, 9923, pp. 1129-37LancetThe Lancet (London), 383, 1129-37

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Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

• Authors : Hugo Fofo; Vince A. Chiodo; David S. Williams

Subjects: Retinal degeneration; retina; DNA, Complementary

• Source: Gene therapy, vol 20, iss 8Gene therapy

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Modulation of Transgene Expression in Retinal Gene Therapy by Selective Laser Treatment

• Authors : Yossi Mandel; Michael F. Marmor; Philip Huie ...

Subjects: Fluorescence-lifetime imaging microscopy; Transgene; Gene Expression

• Source: Repositório Institucional da UNIFESPUniversidade Federal de São Paulo (UNIFESP)instacron:UNIFESP

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Gene Therapy for Retinal Disease: What Lies Ahead.

• Authors : MacLaren, Robert E.¹

• Source: Ophthalmologica. Sep2015, Vol. 234 Issue 1, p1-5. 5p.

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Effective delivery of large genes to the retina by dual AAV vectors

• Authors : Andrea Sommella; Elena Marrocco; Giulia Cesi ...

Subjects: retina; Genetic enhancement; viruses

• Source: EMBO Molecular Medicine

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Retinal transduction profiles by high-capacity viral vectors.

• Authors : Puppo A; Telethon Institute of Genetics and Medicine (TIGEM), Naples, Italy.; Cesi G

Subjects: Dependovirus/Dependovirus/Dependovirus/*genetics ; Herpesvirus 4, Bovine/Herpesvirus 4, Bovine/Herpesvirus 4, Bovine/*genetics ; Lentivirus/Lentivirus/Lentivirus/*genetics

• Source: Gene therapy [Gene Ther] 2014 Oct; Vol. 21 (10), pp. 855-65. Date of Electronic Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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