AIMC Topic: Gene Transfer Techniques

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Intelligent design of tumor microenvironment-responsive Adeno-associated virus vectors: overcoming delivery barriers and enabling precision therapy.

Medical oncology (Northwood, London, England)
Adeno-associated virus (AAV) has emerged as a pivotal vector for cancer gene therapy due to its low immunogenicity, non-pathogenicity, and sustained transgene expression capacity. However, the heterogeneity and complexity of the tumor microenvironmen...

Enhancing lipid nanoparticles-mediated RNA delivery to glioblastoma via targeted strategies.

Journal of controlled release : official journal of the Controlled Release Society
Glioblastoma (GBM) is an aggressive central nervous system (CNS) malignancy with a poor prognosis and limited responses to conventional therapies. RNA-based therapeutics, with their gene-targeting specificity, present a promising avenue for GBM treat...

Microfluidic-based nanocarriers for overcoming biological barriers in therapeutic delivery systems.

Nanoscale
Microfluidic technologies have revolutionized the synthesis of nanocarriers for drug and gene delivery, providing unparalleled precision and efficiency in the production of therapeutic nanoparticles. This review highlights recent advancements in micr...

A multistep platform identifies spleen-tropic lipid nanoparticles for in vivo T cell-targeted delivery of gene-editing proteins.

Science advances
Lipid nanoparticles (LNPs) are a promising nonviral delivery system for gene-editing proteins, but optimal formulations remain underexplored. Unlike messenger RNA-based approaches, ribonucleoprotein delivery enables immediate genome editing without r...

Adipocyte-selective mRNA lipid nanoparticles for cell programming with machine learning analysis.

Journal of controlled release : official journal of the Controlled Release Society
Adipose tissue plays a crucial role in energy metabolism and endocrine signaling. White adipose tissue (WAT), in particular, is a compelling target for therapeutic interventions in metabolic diseases due to its secretory capacity and abundance. Gene ...

Advancing ocular gene therapy: a machine learning approach to enhance delivery, uptake and gene expression.

Drug discovery today
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...

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...

Optimizing lipid nanoparticles for fetal gene delivery in vitro, ex vivo, and aided with machine learning.

Journal of controlled release : official journal of the Controlled Release Society
There is a clinical need to develop lipid nanoparticles (LNPs) to deliver congenital therapies to the fetus during pregnancy. The aim of these therapies is to restore normal fetal development and prevent irreversible conditions after birth. As a firs...

AGILE platform: a deep learning powered approach to accelerate LNP development for mRNA delivery.

Nature communications
Ionizable lipid nanoparticles (LNPs) are seeing widespread use in mRNA delivery, notably in SARS-CoV-2 mRNA vaccines. However, the expansion of mRNA therapies beyond COVID-19 is impeded by the absence of LNPs tailored for diverse cell types. In this ...

Synergistic Polymer Blending Informs Efficient Terpolymer Design and Machine Learning Discerns Performance Trends for pDNA Delivery.

Bioconjugate chemistry
Cationic polymers offer an alternative to viral vectors in nucleic acid delivery. However, the development of polymer vehicles capable of high transfection efficiency and minimal toxicity has remained elusive, and continued exploration of the vast de...