The discovery of clustered regularly interspaced short palindromic repeat (CRISPR)/ CRISPR-associated (Cas) genome editing systems and their applications in human health and medicine has heralded a new era of biotechnology. However, the delivery of CRISPR therapeutics is arguably the most difficult barrier to overcome for translation to clinical administration. Appropriate delivery methods are required to efficiently and selectively transport all gene editing components to specific target cells and tissues of interest, while minimizing off-target effects. To overcome this challenge, we discuss and critic nanoparticle delivery strategies, focusing on the use of lipid-based and polymeric-based matrices herein.
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http://dx.doi.org/10.1016/j.jddst.2021.102728 | DOI Listing |
Nanomedicine (Lond)
December 2024
Department of Bioengineering, King Fahd University of Petroleum and Minerals (KFUPM), Dhahran, Saudi Arabia.
Leber's congenital amaurosis (LCA) represents a set of rare and pervasive hereditary conditions of the retina that cause severe vision loss starting in early childhood. Targeted treatment intervention has become possible thanks to recent advances in understanding LCA genetic basis. While viral vectors have shown efficacy in gene delivery, they present challenges related to safety, low cargo capacity, and the potential for random genomic integration.
View Article and Find Full Text PDFMethods
December 2024
Unidad de Investigación y Desarrollo en Tecnología Farmacéutica (UNITEFA), CONICET and Departamento de Ciencias Farmacéuticas, Facultad de Ciencias Químicas, Universidad Nacional de Córdoba, Ciudad Universitaria, Córdoba 5000, Argentina. Electronic address:
The administration of corticosteroids is the first-line treatment of the clinical conditions with ocular inflammation. Nonetheless, ocular physiological mechanisms, anatomical barriers and corticosteroid properties prevent it from reaching the target site. Thus, frequent topical administered doses or ocular injections are required, leading to a higher risk of adverse events and poor patient compliance.
View Article and Find Full Text PDFActa Pharm Sin B
November 2024
Shanghai Skin Disease Hospital, Tongji University School of Medicine, Shanghai 200443, China.
About 40% of approved drugs and nearly 90% of drug candidates are poorly water-soluble drugs. Low solubility reduces the drugability. Effectively improving the solubility and bioavailability of poorly water-soluble drugs is a critical issue that needs to be urgently addressed in drug development and application.
View Article and Find Full Text PDFNanomedicine
November 2024
Department of Pharmaceutical Sciences and the Biointerfaces Institute, University of Michigan, Ann Arbor, MI 48109, USA. Electronic address:
Adv Healthc Mater
November 2024
Department of Organic and Macromolecular Chemistry, Ghent University, Krijgslaan 281, Gent, 9000, Belgium.
Management of hard-to-heal wounds often requires specialized care that surpasses the capabilities of conventional treatments. Even the most advanced commercial products lack the functionality to meet the needs of hard-to-heal wounds, especially those complicated by active infection, extreme bleeding, and chronic inflammation. The review explores how supramolecular nanovesicles and nanoparticles-such as dendrimers, micelles, polymersomes, and lipid-based nanocarriers-can be key to introducing advanced wound healing and monitoring properties to address the complex needs of hard-to-heal wounds.
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