Visual Field Mean Deviation Slopes: A Potential Primary Endpoint for Clinical Trials in Glaucoma Neuroprotection DOI Creative Commons

Carlos Gustavo De Moraes,

Keith J. Lane,

Xiao Wang

et al.

Research Square (Research Square), Journal Year: 2022, Volume and Issue: unknown

Published: Nov. 11, 2022

Abstract The purpose of this retrospective, longitudinal study is to evaluate the relationship between MD slope from visual field tests collected over a short period time (2 years) and current United States’ Food Drug Administration (FDA) recommended endpoints for outcomes. If correlation strong highly predictive, clinical trials employing slopes as primary could be employed in neuroprotection with shorter duration help expedite development novel IOP-independent therapies. Visual patients or suspected glaucoma were selected an academic institution evaluated based on two functional progression endpoints: 1) five more locations worsening by at least 7 dB, 2) 5 test upon GCP algorithm. A total 467 (76.6%) 472 (77.4%) eyes reached Endpoints B, respectively, any point time. 359 (58.8%) progressed faster than 0 dB/year, 278 (45.5%) -0.5 200 (32.7%) -1.0 dB/year. It was found that experiencing rapid 24-2 2-year likely reach one FDA accepted during soon after period.

Language: Английский

Gene therapies and gene product-based drug candidates for normalizing and preserving tissue functions in animal models of ocular hypertension and glaucoma DOI
Najam A. Sharif

Molecular Aspects of Medicine, Journal Year: 2023, Volume and Issue: 94, P. 101218 - 101218

Published: Nov. 15, 2023

Language: Английский

Citations

2

Eye Disease Genetics and Therapeutics DOI
Najam A. Sharif,

Nozhat Choudry,

Saima D. Chaudhry

et al.

Essentials in ophthalmology/Essentials ophthalmology, Journal Year: 2024, Volume and Issue: unknown, P. 225 - 255

Published: Jan. 1, 2024

Language: Английский

Citations

0

Transcriptome Study of rd1Mouse Brain and Association with Parkinson’s Disease DOI Creative Commons
Ying Zhou,

Yuwei Yang,

Ting Qi

et al.

ACS Omega, Journal Year: 2024, Volume and Issue: 9(24), P. 25756 - 25765

Published: June 6, 2024

Degeneration of the retina is intrinsically associated with pathogenesis and progression neurodegenerative diseases. However, cellular molecular mechanisms underlying association between neurodegeneration retinal degeneration are still under exploration due to complexity connectivity network nervous system. In this study, RNA-seq data from brains model retinitis pigmentosa (RP) mice previously studied Parkinson's disease (PD) were analyzed explore commonalities degenerative Differentially expressed genes in RP compared disease-related intersecting identified, including Cnr1 Septin14. These verified by quantitative real-time reverse transcription PCR Western blotting experiments. The key proteins CNR1 SEPTIN14 found be potential cotherapeutic targets for disease. conclusion, understanding diseases processes brain will not only facilitate interpretation pathomechanisms but also contribute early diagnosis development new therapeutic strategies.

Language: Английский

Citations

0

Emerging Gene Therapy Technologies for Retinal Ganglion Cell Neuroprotection DOI

David K. Camacho,

Cammille C. Go, Brahim Chaqour

et al.

Journal of Neuro-Ophthalmology, Journal Year: 2023, Volume and Issue: unknown

Published: July 12, 2023

Abstract Optic neuropathies encompass a breadth of diseases that ultimately result in dysfunction and/or loss retinal ganglion cells (RGCs). Although visual impairment from optic is common, there lack effective clinical treatments. Addressing critical need for novel interventions, preclinical studies have been generating growing body evidence identify promising new drug-based and cell-based therapies. Gene therapy another emerging therapeutic field offers the potential specifically robustly increasing long-term RGC survival neuropathies. additional benefits driving improvements following single treatment administration, it can be designed to target variety pathways may involved individual or across multiple etiologies. This review explores history gene therapy, fundamentals its application, development technology as relates

Language: Английский

Citations

1

Visual Field Mean Deviation Slopes: A Potential Primary Endpoint for Clinical Trials in Glaucoma Neuroprotection DOI Creative Commons

Carlos Gustavo De Moraes,

Keith J. Lane,

Xiao Wang

et al.

Research Square (Research Square), Journal Year: 2022, Volume and Issue: unknown

Published: Nov. 11, 2022

Abstract The purpose of this retrospective, longitudinal study is to evaluate the relationship between MD slope from visual field tests collected over a short period time (2 years) and current United States’ Food Drug Administration (FDA) recommended endpoints for outcomes. If correlation strong highly predictive, clinical trials employing slopes as primary could be employed in neuroprotection with shorter duration help expedite development novel IOP-independent therapies. Visual patients or suspected glaucoma were selected an academic institution evaluated based on two functional progression endpoints: 1) five more locations worsening by at least 7 dB, 2) 5 test upon GCP algorithm. A total 467 (76.6%) 472 (77.4%) eyes reached Endpoints B, respectively, any point time. 359 (58.8%) progressed faster than 0 dB/year, 278 (45.5%) -0.5 200 (32.7%) -1.0 dB/year. It was found that experiencing rapid 24-2 2-year likely reach one FDA accepted during soon after period.

Language: Английский

Citations

0