AIMC Topic: Gene Transfer Techniques

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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...

Machine learning to determine optimal conditions for controlling the size of elastin-based particles.

Scientific reports
This paper evaluates the aggregation behavior of a potential drug and gene delivery system that combines branched polyethyleneimine (PEI), a positively-charged polyelectrolyte, and elastin-like polypeptide (ELP), a recombinant polymer that exhibits l...

Super-Soft and Super-Elastic DNA Robot with Magnetically Driven Navigational Locomotion for Cell Delivery in Confined Space.

Angewandte Chemie (International ed. in English)
Soft organisms such as earthworms can access confined, narrow spaces, inspiring scientists to fabricate soft robots for in vivo manipulation of cells or tissues and minimally invasive surgery. We report a super-soft and super-elastic magnetic DNA hyd...

Synthesis of Water-Soluble Imidazolium Polyesters as Potential Nonviral Gene Delivery Vehicles.

Biomacromolecules
The inherent hydrolytic reactivity of polyesters renders them excellent candidates for a variety of biomedical applications. Incorporating ionic groups further expands their potential impact, encompassing charge-dependent function such as deoxyribonu...

Preparation of Concentrated Chitosan/DNA Nanoparticle Formulations by Lyophilization for Gene Delivery at Clinically Relevant Dosages.

Journal of pharmaceutical sciences
Chitosan/DNA polyplexes have been optimized for efficient and safe in vitro and in vivo gene delivery. Clinical application of this technology requires the development of formulations with higher concentrations to reach therapeutic dosages. Polyplexe...

Computer-Assisted Transgenesis of Caenorhabditis elegans for Deep Phenotyping.

Genetics
A major goal in the study of human diseases is to assign functions to genes or genetic variants. The model organism Caenorhabditis elegans provides a powerful tool because homologs of many human genes are identifiable, and large collections of geneti...

Revolution of AAV in Drug Discovery: From Delivery System to Clinical Application.

Journal of medical virology
Adeno-associated virus (AAV) is a non-enveloped DNA virus infecting a wide variety of species, tissues, and cell types, which is recognized as a safe and effective method for delivering therapeutic transgenes. AAV vector is the most popular viral gen...

Lipid nanoparticle (LNP) mediated mRNA delivery in neurodegenerative diseases.

Journal of controlled release : official journal of the Controlled Release Society
Neurodegenerative diseases (NDD) are characterized by the progressive loss of neurons and the impairment of cellular functions. Messenger RNA (mRNA) has emerged as a promising therapy for treating NDD, as it can encode missing or dysfunctional protei...

Machine-learning-guided Directed Evolution for AAV Capsid Engineering.

Current pharmaceutical design
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...

Smart Micro/Nano-robotic Systems for Gene Delivery.

Current gene therapy
BACKGROUND: Small scale robotics have attracted growing attention for the prospect of targeting and accessing cell-sized sites, necessary for high precision biomedical applications and drug/gene delivery. The loss of controlled gene therapy, inducing...