scholarly journals Nanoparticle Delivery of CRISPR/Cas9 for Genome Editing

2021 ◽  
Vol 12 ◽  
Author(s):  
Li Duan ◽  
Kan Ouyang ◽  
Xiao Xu ◽  
Limei Xu ◽  
Caining Wen ◽  
...  
Keyword(s):  
System Development ◽  
Delivery Systems ◽  
Inorganic Nanoparticles ◽  
Great Promise ◽  
The Novel ◽  
Disease Treatment ◽  
Nanoparticle Delivery ◽  
Promising Tool ◽  
Short Palindromic Repeat ◽  
Cas Gene

The emerging clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated system (Cas) gene-editing system represents a promising tool for genome manipulation. However, its low intracellular delivery efficiency severely compromises its use and potency for clinical applications. Nanocarriers, such as liposomes, polymers, and inorganic nanoparticles, have shown great potential for gene delivery. The remarkable development of nanoparticles as non-viral carriers for the delivery of the CRISPR/Cas9 system has shown great promise for therapeutic applications. In this review, we briefly summarize the delivery components of the CRISPR/Cas9 system and report on the progress of nano-system development for CRISPR/Cas9 delivery. We also compare the advantages of various nano-delivery systems and their applications to deliver CRISPR/Cas9 for disease treatment. Nano-delivery systems can be modified to fulfill the tasks of targeting cells or tissues. We primarily emphasize the novel exosome-based CRISPR/Cas9 delivery system. Overall, we review the challenges, development trends, and application prospects of nanoparticle-based technology for CRISPR/Cas9 delivery.

scholarly journals Applications and challenges of CRISPR-Cas gene-editing to disease treatment in clinics

2021 ◽  
Author(s):  
Wenyi Liu ◽  
Luoxi Li ◽  
Jianxin Jiang ◽  
Min Wu ◽  
Ping Lin
Keyword(s):  
Gene Editing ◽  
Hereditary Diseases ◽  
Laboratory Research ◽  
Great Promise ◽  
Clinical Tests ◽  
Disease Treatment ◽  
Relative Difficulty ◽  
Agricultural Engineering ◽  
Target Effects ◽  
Cas Gene

Abstract Clustered regularly interspaced short palindromic repeats-CRISPR associated systems (Cas) are efficient tools for targeting specific genes for laboratory research, agricultural engineering, biotechnology, and human disease treatment. Cas9, by far the most extensively used gene-editing nuclease, has shown great promise for the treatment of hereditary diseases, viral infection, cancers and so on. Recent reports have revealed that some other types of CRISPR-Cas systems may also have surprising potential to join the fray as gene-editing tools for various applications. Despite the fast progress in basic researches and clinical tests, some underlying problems present continuous, significant challenges, such as editing efficiency, relative difficulty in delivery, off-target effects, immunogenicity, etc. This article summarizes the applications of CRISPR-Cas from bench to bedside and highlights the current obstacles that may limit the usage of CRISPR-Cas systems as gene-editing tool-kits in precision medicine and offer some viewpoints that may help to tackle these challenges and facilitate technical development. CRISPR-Cas systems, as a powerful gene-editing approach, will offer great hopes in clinical treatments for many individuals with currently incurable diseases.

scholarly journals Improving the Utility of a Dynorphin Peptide Analogue Using Mannosylated Glycoliposomes

2021 ◽  
Vol 22 (15) ◽  
pp. 7996
Author(s):  
Jordan D. Lewicky ◽  
Nya L. Fraleigh ◽  
Alexandrine L. Martel ◽  
Thi M.-D. Nguyen ◽  
Peter W. Schiller ◽  
...  
Keyword(s):  
Targeted Delivery ◽  
Delivery Systems ◽  
Kappa Opioid Receptor ◽  
Antagonist Activity ◽  
Peptide Analogue ◽  
Nanoparticle Delivery ◽  
The Central Nervous System ◽  
Metabolic Degradation ◽  
Distribution Studies

Peptide therapeutics offer numerous advantages in the treatment of diseases and disorders of the central nervous system (CNS). However, they are not without limitations, especially in terms of their pharmacokinetics where their metabolic lability and low blood–brain barrier penetration hinder their application. Targeted nanoparticle delivery systems are being tapped for their ability to improve the delivery of therapeutics into the brain non-invasively. We have developed a family of mannosylated glycoliposome delivery systems for targeted drug delivery applications. Herein, we demonstrate via in vivo distribution studies the potential of these glycoliposomes to improve the utility of CNS active therapeutics using dynantin, a potent and selective dynorphin peptide analogue antagonist of the kappa opioid receptor (KOR). Glycoliposomal entrapment protected dynantin against known rapid metabolic degradation and ultimately improved brain levels of the peptide by approximately 3–3.5-fold. Moreover, we linked this improved brain delivery with improved KOR antagonist activity by way of an approximately 30–40% positive modulation of striatal dopamine levels 20 min after intranasal administration. Overall, the results clearly highlight the potential of our glycoliposomes as a targeted delivery system for therapeutic agents of the CNS.

scholarly journals The Nanosystems Involved in Treating Lung Cancer

Life ◽  
2021 ◽  
Vol 11 (7) ◽  
pp. 682
Author(s):  
Andreea Crintea ◽  
Alina Gabriela Dutu ◽  
Gabriel Samasca ◽  
Ioan Alexandru Florian ◽  
Iulia Lupan ◽  
...  
Keyword(s):  
Lung Cancer ◽  
Side Effects ◽  
Chromosomal Rearrangements ◽  
Sirna Delivery ◽  
Delivery Systems ◽  
Inorganic Nanoparticles ◽  
New Methods ◽  
Different Types ◽  
Or Gene ◽  
The Many

Even though there are various types of cancer, this pathology as a whole is considered the principal cause of death worldwide. Lung cancer is known as a heterogeneous condition, and it is apparent that genome modification presents a significant role in the occurrence of this disorder. There are conventional procedures that can be utilized against diverse cancer types, such as chemotherapy or radiotherapy, but they are hampered by the numerous side effects. Owing to the many adverse events observed in these therapies, it is imperative to continuously develop new and improved strategies for managing individuals with cancer. Nanomedicine plays an important role in establishing new methods for detecting chromosomal rearrangements and mutations for targeted chemotherapeutics or the local delivery of drugs via different types of nano-particle carriers to the lungs or other organs or areas of interest. Because of the complex signaling pathways involved in developing different types of cancer, the need to discover new methods for prevention and detection is crucial in producing gene delivery materials that exhibit the desired roles. Scientists have confirmed that nanotechnology-based procedures are more effective than conventional chemotherapy or radiotherapy, with minor side effects. Several nanoparticles, nanomaterials, and nanosystems have been studied, including liposomes, dendrimers, polymers, micelles, inorganic nanoparticles, such as gold nanoparticles or carbon nanotubes, and even siRNA delivery systems. The cytotoxicity of such nanosystems is a debatable concern, and nanotechnology-based delivery systems must be improved to increase the bioavailability, biocompatibility, and safety profiles, since these nanosystems boast a remarkable potential in many biomedical applications, including anti-tumor activity or gene therapy. In this review, the nanosystems involved in treating lung cancer and its associated challenges are discussed.

scholarly journals Formulation of stabilizer-free, nontoxic PLGA and elastin-PLGA nanoparticle delivery systems

2021 ◽  
Vol 597 ◽  
pp. 120340
Author(s):  
Zachary R. Stromberg ◽  
M. Lisa Phipps ◽  
Harsha D. Magurudeniya ◽  
Christine A. Pedersen ◽  
Trideep Rajale ◽  
...  
Keyword(s):  
Delivery Systems ◽  
Nanoparticle Delivery ◽  
Plga Nanoparticle

scholarly journals Microparticles, Microspheres, and Microcapsules for Advanced Drug Delivery

2019 ◽  
Vol 87 (3) ◽  
pp. 20 ◽  
Author(s):  
Miléna Lengyel ◽  
Nikolett Kállai-Szabó ◽  
Vince Antal ◽  
András József Laki ◽  
István Antal
Keyword(s):  
Drug Delivery ◽  
Drug Delivery Systems ◽  
Freeze Drying ◽  
Delivery Systems ◽  
The Novel ◽  
Practical Applications ◽  
Inhaled Insulin ◽  
Formulation Factors ◽  
Novel Drug ◽  
Significant Production

Microparticles, microspheres, and microcapsules are widely used constituents of multiparticulate drug delivery systems, offering both therapeutic and technological advantages. Microparticles are generally in the 1–1000 µm size range, serve as multiunit drug delivery systems with well-defined physiological and pharmacokinetic benefits in order to improve the effectiveness, tolerability, and patient compliance. This paper reviews their evolution, significance, and formulation factors (excipients and procedures), as well as their most important practical applications (inhaled insulin, liposomal preparations). The article presents the most important structures of microparticles (microspheres, microcapsules, coated pellets, etc.), interpreted with microscopic images too. The most significant production processes (spray drying, extrusion, coacervation, freeze-drying, microfluidics), the drug release mechanisms, and the commonly used excipients, the characterization, and the novel drug delivery systems (microbubbles, microsponges), as well as the preparations used in therapy are discussed in detail.

Insights into the novel three ‘D's of epilepsy treatment: drugs, delivery systems and devices

2010 ◽  
Vol 15 (17-18) ◽  
pp. 717-732 ◽  
Author(s):  
Shadab A. Pathan ◽  
Gaurav K. Jain ◽  
Sohail Akhter ◽  
Divya Vohora ◽  
Farhan J. Ahmad ◽  
...  
Keyword(s):  
Delivery Systems ◽  
The Novel ◽  
Epilepsy Treatment

scholarly journals CRISPR base editors: genome editing without double-stranded breaks

2018 ◽  
Vol 475 (11) ◽  
pp. 1955-1964 ◽  
Author(s):  
Ayman Eid ◽  
Sahar Alshareef ◽  
Magdy M. Mahfouz
Keyword(s):  
Genome Editing ◽  
Dna Sequences ◽  
Genetic Diseases ◽  
Great Promise ◽  
Cytidine Deaminases ◽  
Strand Breaks ◽  
Base Editing ◽  
Potential Applications ◽  
Short Palindromic Repeat ◽  
Basic Biology

The CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9 adaptive immunity system has been harnessed for genome editing applications across eukaryotic species, but major drawbacks, such as the inefficiency of precise base editing and off-target activities, remain. A catalytically inactive Cas9 variant (dead Cas9, dCas9) has been fused to diverse functional domains for targeting genetic and epigenetic modifications, including base editing, to specific DNA sequences. As base editing does not require the generation of double-strand breaks, dCas9 and Cas9 nickase have been used to target deaminase domains to edit specific loci. Adenine and cytidine deaminases convert their respective nucleotides into other DNA bases, thereby offering many possibilities for DNA editing. Such base-editing enzymes hold great promise for applications in basic biology, trait development in crops, and treatment of genetic diseases. Here, we discuss recent advances in precise gene editing using different platforms as well as their potential applications in basic biology and biotechnology.

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