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Toward personalized medicine in Bardet-Biedl syndrome
- Source :
- Personalized medicine. 14(5)
- Publication Year :
- 2018
-
Abstract
- Personalized medicine is becoming routine in the treatment of common diseases such as cancer, but has lagged behind in the field of rare diseases. It is currently in the early stages for the treatment of Bardet–Biedl syndrome. Advances in the understanding of ciliary biology and diagnostic techniques have opened up the prospect of treating BBS in a patient-specific manner. Owing to their structure and function, cilia provide an attractive therapeutic target and genetic therapies are being explored in ciliopathy treatment. Promising avenues include gene therapy, gene editing techniques and splice-correcting and read-through therapies. Targeted drug design has been successful in the treatment of genetic disease and research is underway in the discovery of known and novel drugs to treat Bardet–Biedl syndrome.
- Subjects :
- 0301 basic medicine
congenital, hereditary, and neonatal diseases and abnormalities
Disease
Bioinformatics
03 medical and health sciences
0302 clinical medicine
Genome editing
Bardet–Biedl syndrome
Medicine
Humans
Cilia
Precision Medicine
Bardet-Biedl Syndrome
Pharmacology
business.industry
General Medicine
Genetic Therapy
medicine.disease
Precision medicine
Ciliopathy
030104 developmental biology
Pharmacogenetics
Pharmacogenomics
030221 ophthalmology & optometry
Molecular Medicine
Personalized medicine
business
Subjects
Details
- ISSN :
- 1744828X
- Volume :
- 14
- Issue :
- 5
- Database :
- OpenAIRE
- Journal :
- Personalized medicine
- Accession number :
- edsair.doi.dedup.....146956a28e9cec274ed923e64a423d91