Gene therapy enables the introduction of nucleic acids like DNA and RNA into host cells, and is expected to revolutionize the treatment of a wide range of diseases. This growth has been further accelerated by the discovery of CRISPR/Cas technology, which allows accurate genomic editing in a broad range of cells and organisms . Despite many advances in gene delivery and the development of various viral and non-viral gene delivery vectors, the lack of highly efficient non-viral systems with low cellular toxicity remains a challenge. The application of cutting-edge technologies such as artificial intelligence (AI) has great potential to find new paradigms to solve this issue. Herein, we review AI and its major subfields including machine learning (ML), neural networks (NNs), expert systems, deep learning (DL), computer vision and robotics. We discuss the potential of AI-based models and algorithms in the design of targeted gene delivery vehicles capable of crossing extracellular and intracellular barriers by viral mimicry strategies. We finally discuss the role of AI in improving the function of CRISPR/Cas systems, developing novel nanobots, and mRNA vaccine carriers.
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http://dx.doi.org/10.1016/j.nantod.2022.101665 | DOI Listing |
Cytotherapy
December 2024
Molecular and Clinical Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, Maryland, USA.
Sickle cell disease (SCD) is the most common inherited blood disorder worldwide, impacting millions and imposing severe healthcare challenges, particularly in resource-limited regions. Current treatments have variable efficacy and require lifelong adherence. Allogeneic Hematopoietic Stem Cell Transplantation can be curative but comes with significant side effects and limited donor availability limits its widespread applicability.
View Article and Find Full Text PDFMembranes (Basel)
December 2024
Department of Physical Chemistry, Faculty of Chemistry and Pharmacy, Sofia University St. Kliment Ohridski, 1 "James Bourchier" Blvd., 1164 Sofia, Bulgaria.
Poly(butyl cyanoacrylate) (PBCA) nanoparticles have numerous applications, including drug and gene delivery, molecular imaging, and cancer therapy. To uncover the molecular mechanisms underlying their interactions with cell membranes, we utilized a Langmuir monolayer as a model membrane system. This approach enabled us to investigate the processes of penetration and reorganization of PBCA nanoparticles when deposited in a phospholipid monolayer subphase.
View Article and Find Full Text PDFInvest Ophthalmol Vis Sci
December 2024
Department of Biomedicine, Aarhus University, Aarhus C, Denmark.
Purpose: This review explores the role of pigment epithelium-derived factor (PEDF) in retinal degenerative and vascular disorders and assesses its potential both as an adjunct to established vascular endothelial growth factor inhibiting treatments for retinal vascular diseases and as a neuroprotective therapeutic agent.
Methods: A comprehensive literature review was conducted, focusing on the neuroprotective and anti-angiogenic properties of PEDF. The review evaluated its effects on retinal health, its dysregulation in ocular disorders, and its therapeutic application in preclinical models.
J Pers Med
December 2024
Division of Vascular and Endovascular Surgery, Mayo Clinic, Jacksonville, FL 32224, USA.
Abdominal aortic aneurysm (AAA) is a significant vascular condition characterized by the dilation of the abdominal aorta, presenting a substantial risk of rupture and associated high mortality rates. Current management strategies primarily rely on aneurysm diameter and growth rates to predict rupture risk and determine the timing of surgical intervention. However, this approach has limitations, as ruptures can occur in smaller AAAs below surgical thresholds, and many large AAAs remain stable without intervention.
View Article and Find Full Text PDFCirc Res
December 2024
Cardiovascular Research Center, Massachusetts General Hospital, Boston. (C.C., P.X., Z.Y., Y.S., E.S.L., J.D.R., M.C.H.).
Background: Preeclampsia is a hypertensive disorder of pregnancy characterized by systemic endothelial dysfunction. The pathophysiology of preeclampsia remains incompletely understood. This study used human venous endothelial cell (EC) transcriptional profiling to investigate potential novel mechanisms underlying EC dysfunction in preeclampsia.
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