scholarly journals Viral Delivery Systems for CRISPR

Viruses ◽  
2019 ◽  
Vol 11 (1) ◽  
pp. 28 ◽  
Author(s):  
Christine L. Xu ◽  
Merry Z. C. Ruan ◽  
Vinit B. Mahajan ◽  
Stephen H. Tsang
Keyword(s):  
Animal Models ◽  
Precision Medicine ◽  
Viral Vectors ◽  
Delivery Systems ◽  
Treatment Targets ◽  
Disease Mechanisms

The frontiers of precision medicine have been revolutionized by the development of Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR)/Cas9 as an editing tool. CRISPR/Cas9 has been used to develop animal models, understand disease mechanisms, and validate treatment targets. In addition, it is regarded as an effective tool for genome surgery when combined with viral delivery vectors. In this article, we will explore the various viral mechanisms for delivering CRISPR/Cas9 into tissues and cells, as well as the benefits and drawbacks of each method. We will also review the history and recent development of CRISPR and viral vectors and discuss their applications as a powerful tool in furthering our exploration of disease mechanisms and therapies.

scholarly journals Animal Models Are Valid to Uncover Disease Mechanisms

PLoS Genetics ◽  
2016 ◽  
Vol 12 (5) ◽  
pp. e1006013 ◽  
Author(s):  
Wolfgang Wurst
Keyword(s):  
Animal Models ◽  
Disease Mechanisms

scholarly journals Topical Drug Delivery to the Posterior Segment of the Eye

Pharmaceutics ◽  
2022 ◽  
Vol 14 (1) ◽  
pp. 134
Author(s):  
Marina Löscher ◽  
Chiara Seiz ◽  
José Hurst ◽  
Sven Schnichels
Keyword(s):  
Drug Delivery ◽  
Animal Models ◽  
Treatment Option ◽  
Topical Treatment ◽  
Delivery Systems ◽  
Ocular Drug Delivery ◽  
Posterior Segment ◽  
Retinal Diseases ◽  
Topical Drug Delivery ◽  
Ocular Pharmacokinetics

Topical drug delivery to the posterior segment of the eye is a very complex challenge. However, topical delivery is highly desired, to achieve an easy-to-use treatment option for retinal diseases. In this review, we focus on the drug characteristics that are relevant to succeed in this challenge. An overview on the ocular barriers that need to be overcome and some relevant animal models to study ocular pharmacokinetics are given. Furthermore, a summary of substances that were able to reach the posterior segment after eye drop application is provided, as well as an outline of investigated delivery systems to improve ocular drug delivery. Some promising results of substances delivered to the retina suggest that topical treatment of retinal diseases might be possible in the future, which warrants further research.

scholarly journals Modeling Rare Human Disorders in Mice: The Finnish Disease Heritage

Cells ◽  
2021 ◽  
Vol 10 (11) ◽  
pp. 3158
Author(s):  
Tomáš Zárybnický ◽  
Anne Heikkinen ◽  
Salla M. Kangas ◽  
Marika Karikoski ◽  
Guillermo Antonio Martínez-Nieto ◽  
...  
Keyword(s):  
Human Physiology ◽  
Animal Models ◽  
Mouse Models ◽  
Rare Diseases ◽  
Disease Modeling ◽  
Common Disease ◽  
Molecular Pathways ◽  
Finnish Population ◽  
Disease Mechanisms ◽  
Human Disorders

The modification of genes in animal models has evidently and comprehensively improved our knowledge on proteins and signaling pathways in human physiology and pathology. In this review, we discuss almost 40 monogenic rare diseases that are enriched in the Finnish population and defined as the Finnish disease heritage (FDH). We will highlight how gene-modified mouse models have greatly facilitated the understanding of the pathological manifestations of these diseases and how some of the diseases still lack proper models. We urge the establishment of subsequent international consortiums to cooperatively plan and carry out future human disease modeling strategies. Detailed information on disease mechanisms brings along broader understanding of the molecular pathways they act along both parallel and transverse to the proteins affected in rare diseases, therefore also aiding understanding of common disease pathologies.

Adeno-Associated Viral Vectors for Gene Transfer and Gene Therapy

1999 ◽  
Vol 380 (6) ◽  
Author(s):  
H. Büeler
Keyword(s):  
Gene Expression ◽  
Gene Therapy ◽  
Gene Transfer ◽  
Gene Delivery ◽  
Viral Vectors ◽  
Delivery Systems ◽  
Adeno Associated Virus ◽  
Viral Genes ◽  
Virus Recombinant

AbstractAdeno-associated virus (AAV) is a defective, non-pathogenic human parvovirus that depends for growth on coinfection with a helper adenovirus or herpes virus. Recombinant adeno-associated viruses (rAAVs) have attracted considerable interest as vectors for gene therapy. In contrast to other gene delivery systems, rAAVs lack all viral genes and show long-term gene expression

NON-CONDENSING POLYMERIC GENE DELIVERY SYSTEMS: PRINCIPLES AND APPLICATIONS

Nano LIFE ◽  
2010 ◽  
Vol 01 (03n04) ◽  
pp. 219-237 ◽  
Author(s):  
SHARDOOL JAIN ◽  
HUSAIN ATTARWALA ◽  
MANSOOR AMIJI
Keyword(s):  
Gene Delivery ◽  
Targeted Delivery ◽  
Viral Vectors ◽  
Transfection Efficiency ◽  
Contemporary Literature ◽  
Delivery Systems ◽  
Expression Of Genes ◽  
Cytoplasmic Dna ◽  
Polymeric Delivery ◽  
Systemic Gene Delivery

Gene therapy holds tremendous promise in prevention and treatment of diseases as the approach is based on regulating the expression of genes that are responsible for pathological conditions. The biggest bottleneck for gene delivery has been the development of safe and efficacious delivery systems. Although non-viral vectors are considered as much safer options than their viral counterparts, they suffer from low transfection efficiency. In this review, we highlight the role of non-condensing polymeric delivery systems for oral and systemic gene delivery. Using evidence from contemporary literature, non-condensing polymeric microparticle and nanoparticle systems afford physical encapsulation of the nucleic acid construct and can be engineered for targeted delivery to tissues and cells. Additionally, these systems have shown less toxicity and afford sustained cytoplasmic DNA delivery for efficient nuclear uptake and transfection for both DNA vaccines and therapeutic genes.

scholarly journals Intratracheal aerosolization of viral vectors to newborn pig airways

BioTechniques ◽  
2020 ◽  
Vol 68 (5) ◽  
pp. 235-239
Author(s):  
Ashley L Cooney ◽  
Patrick L Sinn
Keyword(s):  
Gene Therapy ◽  
Animal Models ◽  
Endotracheal Tube ◽  
Viral Vectors ◽  
Small Animal ◽  
Large Animal ◽  
Large Animal Models ◽  
Airway Diseases ◽  
Efficient Delivery ◽  
Large Animals

Gene therapy for airway diseases requires efficient delivery of nucleic acids to the airways. In small animal models, gene delivery reagents are commonly delivered as a bolus dose. However, large animal models are often more relevant for the transition from preclinical studies to human trials. Aerosolizing viral vectors to the lungs of large animals can maximize anatomical distribution. Here, we describe a technique for aerosolization of viral vectors to the airways of newborn pigs. Briefly, a pig is anesthetized and intubated with an endotracheal tube, and a microsprayer is passed through the endotracheal tube. A fine mist is then sprayed into the distal trachea. Widespread and uniform distribution of transgene expression is critical for developing successful lung gene therapy treatments.

scholarly journals Precision medicine in rare disease: Mechanisms of disparate effects of N -carbamyl- l -glutamate on mutant CPS1 enzymes

2017 ◽  
Vol 120 (3) ◽  
pp. 198-206 ◽  
Author(s):  
Dashuang Shi ◽  
Gengxiang Zhao ◽  
Nicholas Ah Mew ◽  
Mendel Tuchman
Keyword(s):  
Precision Medicine ◽  
Rare Disease ◽  
Disease Mechanisms
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