AIMC Topic: Genetic Therapy

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AI-driven innovations in smart multifunctional nanocarriers for drug and gene delivery: A mini-review.

Critical reviews in oncology/hematology
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
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)
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
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
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
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
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...

Analysis on key parameters in subretinal injection facilitating a predictable and automated robot-assisted treatment in gene therapy.

The international journal of medical robotics + computer assisted surgery : MRCAS
BACKGROUND: Subretinal injection (SRI) has become an important surgical method for treating vitreoretinal diseases. Nevertheless, the optimisation of bleb formation in SRI, for the attainment of desired therapeutic outcomes, still requires further in...

Artificial Intelligence (AI)-Aided Structure Optimization for Enhanced Gene Delivery: The Effect of the Polymer Component Distribution (PCD).

ACS applied materials & interfaces
Gene therapy has emerged as a significant advancement in medicine in recent years. However, the development of effective gene delivery vectors, particularly polymer vectors, remains a significant challenge. Limited understanding of the internal struc...

Overcoming Immunological Challenges Limiting Capsid-Mediated Gene Therapy With Machine Learning.

Frontiers in immunology
A key hurdle to making adeno-associated virus (AAV) capsid mediated gene therapy broadly beneficial to all patients is overcoming pre-existing and therapy-induced immune responses to these vectors. Recent advances in high-throughput DNA synthesis, mu...