Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis

SALSABIL MOHAMED ELBORAIE; Kostas Kafetzis; RAJEEV SHRIVASTAVA; ARISTIDES TAGALAKIS

doi:10.5772/intechopen.92434

Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis

SALSABIL MOHAMED ELBORAIE
, Kostas Kafetzis
, RAJEEV SHRIVASTAVA
, ARISTIDES TAGALAKIS

Research output: Chapter in Book/Report/Conference proceeding › Chapter › peer-review

Abstract

The cystic fibrosis transmembrane conductance regulator (CFTR) gene was discovered just over 30 years ago, and soon after, gene therapy for cystic fibrosis (CF) has been rapidly and continually developing. Recently, novel gene therapy strategies have been developed, including mRNA delivery, genome editing, and mRNA repair; all these strategies are collectively named “genetic medicines.” The last quarter of the century showed a significant boost in the development of viral and nonviral vectors to deliver genetic treatment. This chapter will provide a brief overview of the CFTR gene and its different classes of mutations as well as a review of the different genetic therapeutic options that are under research. Later in this chapter, drugs that target different CFTR mutation classes and are currently approved to treat CF patients will be briefly presented.

Original language	English
Title of host publication	Cystic Fibrosis - Facts, Management and Advances
Subtitle of host publication	Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis
Editors	Prashant Mohite
Publisher	IntechOpen
Pages	142-163
Number of pages	21
ISBN (Electronic)	978-1-83881-075-7
ISBN (Print)	978-1-83881-073-3
DOIs	https://doi.org/10.5772/intechopen.92434
Publication status	Published - 22 Apr 2021

Publication series

Name	Cystic Fibrosis - Facts, Management and Advances

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

SDG 3 Good Health and Well-being
SDG 4 Quality Education
SDG 9 Industry, Innovation, and Infrastructure

Keywords

cystic fibrosis
CFTR
gene therapy
CRISPR/Casg
mRNA therapy
gene editing
gene delivery
viral vectors
nonviral vectors
CF animal models
CF drugs

Access to Document

10.5772/intechopen.92434Licence: CC BY

Cite this

ELBORAIE, SALSABIL. MOHAMED., Kafetzis, K., SHRIVASTAVA, RAJEEV., & TAGALAKIS, ARISTIDES. (2021). Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis. In P. Mohite (Ed.), Cystic Fibrosis - Facts, Management and Advances: Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis (pp. 142-163). (Cystic Fibrosis - Facts, Management and Advances). IntechOpen. https://doi.org/10.5772/intechopen.92434

@inbook{1d9b12e192c34d45b996a307ecfffb37,

title = "Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis",

abstract = "The cystic fibrosis transmembrane conductance regulator (CFTR) gene was discovered just over 30 years ago, and soon after, gene therapy for cystic fibrosis (CF) has been rapidly and continually developing. Recently, novel gene therapy strategies have been developed, including mRNA delivery, genome editing, and mRNA repair; all these strategies are collectively named “genetic medicines.” The last quarter of the century showed a significant boost in the development of viral and nonviral vectors to deliver genetic treatment. This chapter will provide a brief overview of the CFTR gene and its different classes of mutations as well as a review of the different genetic therapeutic options that are under research. Later in this chapter, drugs that target different CFTR mutation classes and are currently approved to treat CF patients will be briefly presented.",

keywords = "cystic fibrosis, CFTR, gene therapy, CRISPR/Casg, mRNA therapy, gene editing, gene delivery, viral vectors, nonviral vectors, CF animal models, CF drugs",

author = "ELBORAIE, \{SALSABIL MOHAMED\} and Kostas Kafetzis and RAJEEV SHRIVASTAVA and ARISTIDES TAGALAKIS",

year = "2021",

month = apr,

day = "22",

doi = "10.5772/intechopen.92434",

language = "English",

isbn = "978-1-83881-073-3",

series = "Cystic Fibrosis - Facts, Management and Advances",

publisher = "IntechOpen",

pages = "142--163",

editor = "Mohite, \{Prashant \}",

booktitle = "Cystic Fibrosis - Facts, Management and Advances",

}

ELBORAIE, SALSABILMOHAMED, Kafetzis, K, SHRIVASTAVA, RAJEEV & TAGALAKIS, ARISTIDES 2021, Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis. in P Mohite (ed.), Cystic Fibrosis - Facts, Management and Advances: Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis. Cystic Fibrosis - Facts, Management and Advances, IntechOpen, pp. 142-163. https://doi.org/10.5772/intechopen.92434

Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis. / ELBORAIE, SALSABIL MOHAMED; Kafetzis, Kostas; SHRIVASTAVA, RAJEEV et al.
Cystic Fibrosis - Facts, Management and Advances: Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis. ed. / Prashant Mohite. IntechOpen, 2021. p. 142-163 (Cystic Fibrosis - Facts, Management and Advances).

Research output: Chapter in Book/Report/Conference proceeding › Chapter › peer-review

TY - CHAP

T1 - Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis

AU - ELBORAIE, SALSABIL MOHAMED

AU - Kafetzis, Kostas

AU - SHRIVASTAVA, RAJEEV

AU - TAGALAKIS, ARISTIDES

PY - 2021/4/22

Y1 - 2021/4/22

N2 - The cystic fibrosis transmembrane conductance regulator (CFTR) gene was discovered just over 30 years ago, and soon after, gene therapy for cystic fibrosis (CF) has been rapidly and continually developing. Recently, novel gene therapy strategies have been developed, including mRNA delivery, genome editing, and mRNA repair; all these strategies are collectively named “genetic medicines.” The last quarter of the century showed a significant boost in the development of viral and nonviral vectors to deliver genetic treatment. This chapter will provide a brief overview of the CFTR gene and its different classes of mutations as well as a review of the different genetic therapeutic options that are under research. Later in this chapter, drugs that target different CFTR mutation classes and are currently approved to treat CF patients will be briefly presented.

AB - The cystic fibrosis transmembrane conductance regulator (CFTR) gene was discovered just over 30 years ago, and soon after, gene therapy for cystic fibrosis (CF) has been rapidly and continually developing. Recently, novel gene therapy strategies have been developed, including mRNA delivery, genome editing, and mRNA repair; all these strategies are collectively named “genetic medicines.” The last quarter of the century showed a significant boost in the development of viral and nonviral vectors to deliver genetic treatment. This chapter will provide a brief overview of the CFTR gene and its different classes of mutations as well as a review of the different genetic therapeutic options that are under research. Later in this chapter, drugs that target different CFTR mutation classes and are currently approved to treat CF patients will be briefly presented.

KW - cystic fibrosis

KW - CFTR

KW - gene therapy

KW - CRISPR/Casg

KW - mRNA therapy

KW - gene editing

KW - gene delivery

KW - viral vectors

KW - nonviral vectors

KW - CF animal models

KW - CF drugs

UR - https://www.mendeley.com/catalogue/fcb6b36f-e848-3e5b-be06-40581097ceaf/

U2 - 10.5772/intechopen.92434

DO - 10.5772/intechopen.92434

M3 - Chapter

SN - 978-1-83881-073-3

T3 - Cystic Fibrosis - Facts, Management and Advances

SP - 142

EP - 163

BT - Cystic Fibrosis - Facts, Management and Advances

A2 - Mohite, Prashant

PB - IntechOpen

ER -

ELBORAIE SALSABILMOHAMED, Kafetzis K, SHRIVASTAVA RAJEEV, TAGALAKIS ARISTIDES. Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis. In Mohite P, editor, Cystic Fibrosis - Facts, Management and Advances: Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis. IntechOpen. 2021. p. 142-163. (Cystic Fibrosis - Facts, Management and Advances). doi: 10.5772/intechopen.92434

Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis

Abstract

Publication series

UN SDGs

Keywords

Access to Document

Other files and links

Fingerprint

Cite this