Gene therapeutics and protein development

WEEK

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Gene Therapeutics: Nanoplexes
and Other Carriers
Prof. Amal Ali Elkordy
Professor of Pharmaceutics
e mail:
Office: Dale 104



Slide 1 of ? MPHM??
Gene Module
Therapeutics: Introduction
Nanoplexes and Other Carriers

,WEEK

?9
32 Gene Therapeutics: Nanoplexes and Other
Carriers
Overview
❖ What is Gene Therapy?
❖ What are Human Genetic Diseases?
❖ Principles for gene delivery to cell nuclei
❖ Problematic properties of DNA
❖ DNA carrier systems:
Lipoplexes, Polyplexes and Nanoplexes
Cyclodextrins
Slide
Slide 22 of
of ?20 MPHM??
Gene Module
Therapeutics: Introduction
Nanoplexes and Other Carriers

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32
❖ What is Gene Therapy?
Human genetic disease= result of mutation or
deletion of gene (s)
❖ Aims: To treat the cause rather than the
symptoms of the diseases= caused by single
gene mutation or deletion (e.g. Haemophilia,
Cystic fibrosis, Muscular dystrophy)
❖Treatable acquired diseases= more than one
gene affected and no clear identification of the
position (s) of the defect (e.g. cancer,
Parkinson’s & Alzheimer's disease )
Slide
Slide 33 of
of ?20 MPHM07 MPHM??Gene Module Introduction
Therapeutics: Nanoplexes and Other Carriers

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32 ❖ Delivery of replacement or insertion gene
sequence into cell nucleus
❖ To reach site of action, DNA sequence must pass
a number of compartments.
DNA, RNA, central dogma of molecular biology
DNA= deoxyribonucleic acid, carbohydrate unit
2-D-Deoxyribose
= represents genetic material
RNA= ribonucleic acid, carbohydrate unit 2-D-
Ribose
= essential for biosynthesis of proteins
=different types; here important mRNA
Slide
Slide 44 of
of ?20 MPHM07 MPHM??Gene Module Introduction
Therapeutics: Nanoplexes and Other Carriers