AIMC Topic: Genetic Therapy
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Deep Learning-Based SD-OCT Layer Segmentation Quantifies Outer Retina Changes in Patients With Biallelic RPE65 Mutations Undergoing Gene Therapy.
Investigative ophthalmology & visual science
Jan 2, 2025
PURPOSE: To quantify outer retina structural changes and define novel biomarkers of inherited retinal degeneration associated with biallelic mutations in RPE65 (RPE65-IRD) in patients before and after subretinal gene augmentation therapy with voretig...
[LORENZO'S OIL AND ADRENOLEUKODYSTROPHY EXAMINING AN ARTIFICIAL INTELLIGENCE TOOL INTENDED FOR CONDUCTING LITERATURE SEARCHES AND ANALYSES].
Harefuah
Dec 1, 2024
Adrenoleukodystrophy is a genetic metabolic disorder characterized by a heterogeneous phenotype. Its severe form, known as cerebral adrenoleukodystrophy, involves unpredictable cerebral damage and progressive central nervous system deterioration. Thi...
Machine-learning-guided Directed Evolution for AAV Capsid Engineering.
Current pharmaceutical design
Jan 1, 2024
Target gene delivery is crucial to gene therapy. Adeno-associated virus (AAV) has emerged as a primary gene therapy vector due to its broad host range, long-term expression, and low pathogenicity. However, AAV vectors have some limitations, such as i...
Recent Progress of Machine Learning in Gene Therapy.
Current gene therapy
Jan 1, 2022
With new developments in biomedical technology, it is now a viable therapeutic treatment to alter genes with techniques like CRISPR. At the same time, it is increasingly cheaper to perform whole genome sequencing, resulting in rapid advancement in ge...
The retina revolution: signaling pathway therapies, genetic therapies, mitochondrial therapies, artificial intelligence.
Current opinion in ophthalmology
May 1, 2020
PURPOSE OF REVIEW: The aim of this article is to review and discuss the history, current state, and future implications of promising biomedical offerings in the field of retina.
Recent developments in pediatric retina.
Current opinion in ophthalmology
May 1, 2020
PURPOSE OF REVIEW: Pediatric retina is an exciting, but also challenging field, where patient age and cooperation can limit ease of diagnosis of a broad range of congenital and acquired diseases, inherited retinal degenerations are mostly untreatable...
Omics Data and Artificial Intelligence: New Challenges for Gene Therapy.
Current gene therapy
Jan 1, 2020
Future Vision 2020 and Beyond-5 Critical Trends in Eye Research.
Asia-Pacific journal of ophthalmology (Philadelphia, Pa.)
Jan 1, 2020
Ophthalmology has been at the forefront of many innovations in basic science and clinical research. The randomized prospective multicenter clinical trial, comparative clinical trials, the bench to beside development of diagnostic and therapeutic devi...
Treatment Potential for Macular Cone Vision in Leber Congenital Amaurosis Due to CEP290 or NPHP5 Mutations: Predictions From Artificial Intelligence.
Investigative ophthalmology & visual science
Jun 3, 2019
PURPOSE: To use supervised machine learning to predict visual function from retinal structure in retinitis pigmentosa (RP) and apply these estimates to CEP290- and NPHP5-associated Leber congenital amaurosis (LCA) to determine the potential for funct...
Retinitis Pigmentosa
Retinal Cone Photoreceptor Cells
Calmodulin-Binding Proteins
Male
Leber Congenital Amaurosis
Middle Aged
Genetic Therapy
Mutation
Humans
Adolescent
Cell Cycle Proteins
Visual Fields
Visual Field Tests
Antigens, Neoplasm
Machine Learning
Female
Cytoskeletal Proteins
Color Vision Defects
Neoplasm Proteins
Young Adult
Tomography, Optical Coherence
Adult
Deep Forest-based Prediction of Protein Subcellular Localization.
Current gene therapy
Jan 1, 2018
MOTIVATION: Knowledge of the correct protein subcellular localization is necessary for understanding the function of a protein and revealing the mechanism of many human diseases due to protein subcellular mislocalization, which is required before app...
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