AIMC Topic: Genetic Therapy
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Prediction of Adeno-Associated Virus Fitness with a Protein Language-Based Machine Learning Model.
Human gene therapy
Apr 16, 2025
Adeno-associated virus (AAV)-based therapeutics have the potential to transform the lives of patients by delivering one-time treatments for a variety of diseases. However, a critical challenge to their widespread adoption and distribution is the high...
Advancing ocular gene therapy: a machine learning approach to enhance delivery, uptake and gene expression.
Drug discovery today
Apr 12, 2025
Ocular gene therapy offers a promising approach for treating various eye diseases, centered on the process of transfection, including delivery, cellular uptake and gene expression. This study addresses anatomical and physiological barriers, such as t...
Advances in AAV capsid engineering: Integrating rational design, directed evolution and machine learning.
Molecular therapy : the journal of the American Society of Gene Therapy
Apr 1, 2025
Adeno-associated virus (AAV) has emerged as a highly promising vector for human gene therapy due to its favorable safety profile, versatility, and ability to transduce a wide range of tissues. However, natural AAV serotypes have shortcomings, includi...
AI-driven innovations in smart multifunctional nanocarriers for drug and gene delivery: A mini-review.
Critical reviews in oncology/hematology
Mar 13, 2025
The convergence of artificial intelligence (AI) and nanomedicine has revolutionized the design of smart multifunctional nanocarriers (SMNs) for drug and gene delivery, offering unprecedented precision, efficiency, and personalization in therapeutic a...
Enhancing Functional Protein Design Using Heuristic Optimization and Deep Learning for Anti-Inflammatory and Gene Therapy Applications.
Proteins
Feb 22, 2025
Protein sequence design is a highly challenging task, aimed at discovering new proteins that are more functional and producible under laboratory conditions than their natural counterparts. Deep learning-based approaches developed to address this prob...
Artificial Intelligence-Based Approaches for AAV Vector Engineering.
Advanced science (Weinheim, Baden-Wurttemberg, Germany)
Feb 11, 2025
Adeno-associated virus (AAV) has emerged as a leading vector for gene therapy due to its broad host range, low pathogenicity, and ability to facilitate long-term gene expression. However, AAV vectors face limitations, including immunogenicity and ins...
Protocol for functional screening of CFTR-targeted genetic therapies in patient-derived organoids using DETECTOR deep-learning-based analysis.
STAR protocols
Jan 31, 2025
Here, we present a protocol for the rapid functional screening of gene editing and addition strategies in patient-derived organoids using the deep-learning-based tool DETECTOR (detection of targeted editing of cystic fibrosis transmembrane conductanc...
AI-Based solutions for current challenges in regenerative medicine.
European journal of pharmacology
Oct 24, 2024
The emergence of Artificial Intelligence (AI) and its usage in regenerative medicine represents a significant opportunity that holds the promise of tackling critical challenges and improving therapeutic outcomes. This article examines the ways in whi...
An interpretable data-driven prediction model to anticipate scoliosis in spinal muscular atrophy in the era of (gene-) therapies.
Scientific reports
May 23, 2024
5q-spinal muscular atrophy (SMA) is a neuromuscular disorder (NMD) that has become one of the first 5% treatable rare diseases. The efficacy of new SMA therapies is creating a dynamic SMA patient landscape, where disease progression and scoliosis dev...
Artificial Intelligence and Computational Biology in Gene Therapy: A Review.
Biochemical genetics
Apr 18, 2024
One of the trending fields in almost all areas of science and technology is artificial intelligence. Computational biology and artificial intelligence can help gene therapy in many steps including: gene identification, gene editing, vector design, de...
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