☆ 4.7 Editorial Material

Does systemic inflammation prompt gene therapy uveitis?

MOLECULAR THERAPY (2021)

Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial

Alaknanda Mishra et al.

Summary: This study revealed that patients with XLRS may have a proinflammatory baseline immune phenotype, and intravitreal dosing with AAV8-RS1 leads to systemic immune activation characterized by an increase in activated lymphocytes, macrophages, and proinflammatory cytokines.

MOLECULAR THERAPY (2021)

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Article Cell Biology

Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses

Ying Kai Chan et al.

Summary: The study engineered AAV vectors by incorporating short DNA oligonucleotides to antagonize TLR9 activation, reducing innate immune responses and enhancing gene expression in clinically relevant animal models. The engineered vectors can avoid adverse reactions in some models, demonstrating a potential wider therapeutic window for AAV therapies.

SCIENCE TRANSLATIONAL MEDICINE (2021)

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Review Ophthalmology

Inflammation in Viral Vector-Mediated Ocular Gene Therapy: A Review and Report From a Workshop Hosted by the Foundation Fighting Blindness, 9/2020

Ying Kai Chan et al.

Summary: The workshop focused on discussing intraocular inflammation during gene therapy for inherited retinal diseases, with participants agreeing that both clinical and subclinical inflammation are common during the treatment process. The severity of inflammation is related to vector dose, and further research is needed to understand the relationship between inflammation and treatment efficacy.

TRANSLATIONAL VISION SCIENCE & TECHNOLOGY (2021)

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Review Neurosciences

Microglia versus Monocytes: Distinct Roles in Degenerative Diseases of the Retina

Chen Yu et al.

TRENDS IN NEUROSCIENCES (2020)

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Article Multidisciplinary Sciences

Effects of processing conditions on stability of immune analytes in human blood

Andres Gottfried-Blackmore et al.

SCIENTIFIC REPORTS (2020)

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Article Biotechnology & Applied Microbiology

Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

Catherine Cukras et al.

MOLECULAR THERAPY (2018)

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Article Hematology

The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver

Ashley T. Martino et al.

BLOOD (2011)

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Does systemic inflammation prompt gene therapy uveitis?

相关参考文献

Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial

Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses

Inflammation in Viral Vector-Mediated Ocular Gene Therapy: A Review and Report From a Workshop Hosted by the Foundation Fighting Blindness, 9/2020

Microglia versus Monocytes: Distinct Roles in Degenerative Diseases of the Retina

Effects of processing conditions on stability of immune analytes in human blood

Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver

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