alexa Advantage of Delivering the Gene Therapy Tool (Cas9) as Purified Protein Formulation

Journal of Formulation Science & Bioavailability
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Review Article

Advantage of Delivering the Gene Therapy Tool (Cas9) as Purified Protein Formulation

Doolaanea AA*

Advanced Drug Delivery Lab, Department of Pharmaceutical Technology, Kulliyyah of Pharmacy, International Islamic University Malaysia, 25200 Kuantan, Malaysia

*Corresponding Author:
Doolaanea AA
Advanced Drug Delivery Lab
Department of Pharmaceutical Technology
Kulliyyah of Pharmacy
International Islamic University Malaysia, Malaysia
Tel: +60136238628
E-mail: [email protected]

Received Date: May 02, 2017; Accepted Date: May 15, 2017; Published Date: May 25, 2017

Citation: Doolaanea AA (2017) Advantage of Delivering the Gene Therapy Tool (Cas9) as Purified Protein Formulation. J Formul Sci Bioavailab 1: 111.

Copyright: © 2017 Doolaanea AA. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

 

Abstract

Gene editing permits the alteration of genomic sequences in vitro and in vivo, which could serve as a powerful tool in basic research and human gene therapy. The recent clustered regularly interspaced short palindromic repeat (CRISPR) system has revolutionized the way of gene editing. To be used in human, a suitable in vivo-compatible delivery form and method should be used. The delivery form here is meant ‘what’ is being introduced to the body: plasmid DNA, viral vector, mRNA, purified protein or other while the delivery method is meant ‘how’ the CRISPR system is introduced or administered: lipid transfection, electroporation, nanoparticles, liposomes or others. The brief discussion in this article suggests that delivering the protein component of the CRISPR system as purified protein has several advantages considering different sides like the industrial feasibility and pharmacokinetic/ pharmacodynamic aspects.

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