We use cookies to understand how you use our site and to improve your experience. This includes personalizing content and advertising. To learn more, click here. By continuing to use our site, you accept our use of cookies. Cookie Policy.

Features Partner Sites Information LinkXpress
Sign In
Advertise with Us
LGC Clinical Diagnostics

Download Mobile App




RNA-Based Gene Therapy Developed for Cystic Fibrosis

By LabMedica International staff writers
Posted on 05 Jul 2018
Print article
Image: Structure of the CFTR (cystic fibrosis transmembrane conductance regulator) protein. Mutations of the CFTR gene affecting chloride ion channel function lead to dysregulation of epithelial fluid transport in the lung, pancreas, and other organs, resulting in cystic fibrosis (Photo courtesy of Wikimedia Commons).
Image: Structure of the CFTR (cystic fibrosis transmembrane conductance regulator) protein. Mutations of the CFTR gene affecting chloride ion channel function lead to dysregulation of epithelial fluid transport in the lung, pancreas, and other organs, resulting in cystic fibrosis (Photo courtesy of Wikimedia Commons).
A potential gene therapy approach for treating cystic fibrosis utilizes lipid nanoparticle-delivered chemically modified mRNA.

The promise of gene therapy for the treatment of cystic fibrosis has yet to be fully clinically realized despite years of effort toward correcting the underlying genetic defect in CFTR (cystic fibrosis transmembrane conductance regulator). Mutations in CFTR cause the disease, which is characterized by lung dehydration and mucous buildup that blocks the airway. Nanoparticle delivery of messenger RNA (mRNA) represents a powerful technology for the transfer of genetic material to cells with large, widespread populations, such as airway epithelia.

Investigators at Oregon State University (Portland, USA) and Oregon Health & Science University (Portland, USA) deployed a clinically relevant lipid-based nanoparticle (LNP) for packaging and delivery of large chemically modified CFTR mRNA (cmCFTR) to patient-derived bronchial epithelial cells.

Results published in the June 14, 2018, online edition of the journal Molecular Therapy revealed that this treatment caused in an increase in membrane-localized CFTR and rescue of its primary function as a chloride channel. Furthermore, nasal application of LNP-cmCFTR restored CFTR-mediated chloride secretion to conductive airway epithelia in CFTR knockout mice for at least 14 days. On day three post-transfection, CFTR activity peaked, recovering up to 55% of the net chloride efflux characteristic of healthy mice. This magnitude of response was superior to liposomal CFTR DNA delivery and was comparable with outcomes observed in the currently approved drug ivacaftor.

"It was a platform technology for correcting monogenic disorders and allows the same therapy to be effective for treating all cystic fibrosis patients and these systems can be repeatedly administered to a patient and the effects are reversible if someone needs to stop the therapy for any reason," said senior author Dr. Gaurav Sahay, assistant professor of pharmaceutical sciences at Oregon State University/Oregon Health & Science University.

Related Links:
Oregon State University
Oregon Health & Science University

Platinum Member
COVID-19 Rapid Test
OSOM COVID-19 Antigen Rapid Test
HLX
Complement 3 (C3) Test
GPP-100 C3 Kit
New
Gold Member
TORCH Panel Rapid Test
Rapid TORCH Panel Test

Print article

Channels

Clinical Chemistry

view channel
Image: Reaching speeds up to 6,000 RPM, this centrifuge forms the basis for a new type of inexpensive, POC biomedical test (Photo courtesy of Duke University)

POC Biomedical Test Spins Water Droplet Using Sound Waves for Cancer Detection

Exosomes, tiny cellular bioparticles carrying a specific set of proteins, lipids, and genetic materials, play a crucial role in cell communication and hold promise for non-invasive diagnostics.... Read more

Molecular Diagnostics

view channel
Image: MOF materials efficiently enrich cfDNA and cfRNA in blood through simple operational process (Photo courtesy of Science China Press)

Blood Circulating Nucleic Acid Enrichment Technique Enables Non-Invasive Liver Cancer Diagnosis

The ability to diagnose diseases early can significantly enhance the effectiveness of clinical treatments and improve survival rates. One promising approach for non-invasive early diagnosis is the use... Read more

Hematology

view channel
Image: The low-cost portable device rapidly identifies chemotherapy patients at risk of sepsis (Photo courtesy of 52North Health)

POC Finger-Prick Blood Test Determines Risk of Neutropenic Sepsis in Patients Undergoing Chemotherapy

Neutropenia, a decrease in neutrophils (a type of white blood cell crucial for fighting infections), is a frequent side effect of certain cancer treatments. This condition elevates the risk of infections,... Read more

Pathology

view channel
Image: The OvaCis Rapid Test discriminates benign from malignant epithelial ovarian cysts (Photo courtesy of INEX)

Intra-Operative POC Device Distinguishes Between Benign and Malignant Ovarian Cysts within 15 Minutes

Ovarian cysts represent a significant health issue for women globally, with up to 10% experiencing this condition at some point in their lives. These cysts form when fluid collects within a thin membrane... Read more
Copyright © 2000-2024 Globetech Media. All rights reserved.