Abstract
Cystic Fibrosis (CF) is a serious genetic condition caused by CF transmembrane conductance regulator (CFTR) mutation. CF patients have shortened lifespan due to airway obstruction, infection, and end-stage lung failure. However, recent development in CF therapy suggests a brighter future for CF patients. Targeting specific CFTR mutations aims to potentiate the channel gating activity of impaired CFTR and restore protein trafficking to the plasma membrane. Gene therapy introduces correct CFTR gene into the affected airway epithelium leading to the functional expression of CFTR in CF patients. This review will sum up the current status in CF-cause targeting therapy.
Keywords: CFTR, chloride transporter, corrector, cystic fibrosis, gene therapy, potentiator.
Current Drug Targets
Title:Targeting the Root Cause of Cystic Fibrosis
Volume: 16 Issue: 9
Author(s): Laura Trescott, Joshua Holcomb, Nicholas Spellmon, Cathy Mcleod, Leala Aljehane, Fei Sun, Chunying Li and Zhe Yang
Affiliation:
Keywords: CFTR, chloride transporter, corrector, cystic fibrosis, gene therapy, potentiator.
Abstract: Cystic Fibrosis (CF) is a serious genetic condition caused by CF transmembrane conductance regulator (CFTR) mutation. CF patients have shortened lifespan due to airway obstruction, infection, and end-stage lung failure. However, recent development in CF therapy suggests a brighter future for CF patients. Targeting specific CFTR mutations aims to potentiate the channel gating activity of impaired CFTR and restore protein trafficking to the plasma membrane. Gene therapy introduces correct CFTR gene into the affected airway epithelium leading to the functional expression of CFTR in CF patients. This review will sum up the current status in CF-cause targeting therapy.
Export Options
About this article
Cite this article as:
Trescott Laura, Holcomb Joshua, Spellmon Nicholas, Mcleod Cathy, Aljehane Leala, Sun Fei, Li Chunying and Yang Zhe, Targeting the Root Cause of Cystic Fibrosis, Current Drug Targets 2015; 16 (9) . https://dx.doi.org/10.2174/1389450115999141030144247
DOI https://dx.doi.org/10.2174/1389450115999141030144247 |
Print ISSN 1389-4501 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-5592 |
Call for Papers in Thematic Issues
New drug therapy for eye diseases
Eyesight is one of the most critical senses, accounting for over 80% of our perceptions. Our quality of life might be significantly affected by eye disease, including glaucoma, diabetic retinopathy, dry eye, etc. Although the development of microinvasive ocular surgery reduces surgical complications and improves overall outcomes, medication therapy is ...read more
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
Related Articles
-
Designing and Testing New Therapeutic Modalities for Treatment of Inflammatory Bowel Disease: Role of Experimental Animal Models
Current Medicinal Chemistry - Anti-Inflammatory & Anti-Allergy Agents Enzyme Inhibitors
Current Bioactive Compounds NAMPT in Regulated NAD Biosynthesis and its Pivotal Role in Human Metabolism
Current Medicinal Chemistry Applications of Nuclear Technique to Biological Sciences Labelled Compounds, Radioactive Tracers, and X-Ray Tomography
Current Topics in Medicinal Chemistry New Therapeutic Applications of Phosphodiesterase 5 Inhibitors (PDE5-Is)
Current Medicinal Chemistry Potential Therapeutic Agents Against Par-4 Target for Cancer Treatment: Where Are We Going?
Current Drug Targets Alpha-Emitters for Immuno-Therapy: A Review of Recent Developments from Chemistry to Clinics
Current Topics in Medicinal Chemistry Chemical and physical factors influencing the dynamics of differentiation in embryonic stem cells
Current Stem Cell Research & Therapy Differential Action of Phytochemicals on Platelet Apoptosis: A Biological Overview
Current Medicinal Chemistry A Prospective Study of the Clinical and Demographic Profile of Type 2 Diabetes Mellitus Patients Receiving Antidiabetic Drug Combinations
Current Diabetes Reviews Herpes Virus Entry Mediator, which Decreases in HIV Infection, Can Enhance the Suppressive Activity of Regulatory T Cell and Predict Recovery of CD4+T-cells During HAART
Current HIV Research Nanomedicine as a Strategy for Natural Compound Delivery to Prevent and Treat Cancers
Current Pharmaceutical Design Mechanisms Involved in Metformin Action in the Treatment of Polycystic Ovary Syndrome
Current Pharmaceutical Design Meet Our Editorial Board Member
Anti-Cancer Agents in Medicinal Chemistry Sirtuins: Nodes Connecting Aging, Metabolism and Tumorigenesis
Current Pharmaceutical Design Oncogenic Fusion Tyrosine Kinases as Molecular Targets for Anti-Cancer Therapy
Anti-Cancer Agents in Medicinal Chemistry Human Complex Trait Genetics: Lifting the Lid of the Genomics Toolbox - from Pathways to Prediction
Current Genomics EDITORIAL (Thematic Issue: Emerging Roles of Epithelial-to-Mesenchymal Transition in Tumorigenesis)
Current Cancer Drug Targets Stable Isotope Techniques to Develop and Monitor Nutrition Interventions
Current Nutrition & Food Science 11C-Choline PET/CT based Helical Tomotherapy as Treatment Approach for Bone Metastases in Recurrent Prostate Cancer Patients
Current Radiopharmaceuticals