Pradnya Rajesh Khunte
, Dipali Pagire , Papiha Raut , Akash patil
Cystic fibrosis; cystic fibrosis transmembrane conductance regulator (CFTR), gene editing; nanocarriers
Cystic fibrosis (CF) is a hereditary, multisystemic disease cause by different mutations in the CFTR gene encoding CF transmembrane conductance regulator. CF is mainly characterized by pulmonary dysfunction as a result of deterioration in the mucociliary clearance and anion transport of airways. Mortality is mostly caused by bronchiectasis, bronchiole obstruction, and progressive respiratory dysfunction in the early years of life. Over the last decade, new therapeutic strategies rather than symptomatic treatment have been superposed, such as the small molecule approach, ion channel therapy, and pulmonary gene therapy. Due to considerable progress in the treatment options, CF has become an adult disease rather than a paediatric disease in recent years. Pulmonary gene therapy has gain special attention due to its mutation type independent aspect, therefore being applicable to all CF patients. On the other hand, the major obstacle for cystic fibrosis treatment is to predict the drug response of patients due to genetic complexity and heterogeneity. The advancement of 3D culture systems has made it possible to extrapolate the disease modelling and individual drug response in vitro by making mini adult organs called “organoids” obtained from rectal cell biopsies. In this review, we summarize the advances in the novel therapeutic approaches, clinical interventions, and precision medicine concept for disease.
"Study of Cystic Fibrosis", IJSDR - International Journal of Scientific Development and Research (www.IJSDR.org), ISSN:2455-2631, Vol.7, Issue 11, page no.463 - 474, November-2022, Available :https://ijsdr.org/papers/IJSDR2211074.pdf
Volume 7
Issue 11,
November-2022
Pages : 463 - 474
Paper Reg. ID: IJSDR_202572
Published Paper Id: IJSDR2211074
Downloads: 000347205
Research Area: Pharmacy
Country: Shrirampur, Ahmednagar, Maharashtra, India
ISSN: 2455-2631 | IMPACT FACTOR: 9.15 Calculated By Google Scholar | ESTD YEAR: 2016
An International Scholarly Open Access Journal, Peer-Reviewed, Refereed Journal Impact Factor 9.15 Calculate by Google Scholar and Semantic Scholar | AI-Powered Research Tool, Multidisciplinary, Monthly, Multilanguage Journal Indexing in All Major Database & Metadata, Citation Generator
Publisher: IJSDR(IJ Publication) Janvi Wave
