Next Article in Journal
Metabolic Syndrome and the Risk of Breast Cancer and Subtypes by Race, Menopause and BMI
Previous Article in Journal
The p53 Pathway in Glioblastoma
 
 
Review

Rare Genetic Diseases with Defects in DNA Repair: Opportunities and Challenges in Orphan Drug Development for Targeted Cancer Therapy

Cold Spring Harbor Laboratory, Cold Spring Harbor, New York, NY 11724, USA
*
Authors to whom correspondence should be addressed.
Cancers 2018, 10(9), 298; https://doi.org/10.3390/cancers10090298
Received: 9 July 2018 / Revised: 27 August 2018 / Accepted: 28 August 2018 / Published: 1 September 2018
A better understanding of mechanistic insights into genes and enzymes implicated in rare diseases provide a unique opportunity for orphan drug development. Advances made in identification of synthetic lethal relationships between rare disorder genes with oncogenes and tumor suppressor genes have brought in new anticancer therapeutic opportunities. Additionally, the rapid development of small molecule inhibitors against enzymes that participate in DNA damage response and repair has been a successful strategy for targeted cancer therapeutics. Here, we discuss the recent advances in our understanding of how many rare disease genes participate in promoting genome stability. We also summarize the latest developments in exploiting rare diseases to uncover new biological mechanisms and identify new synthetic lethal interactions for anticancer drug discovery that are in various stages of preclinical and clinical studies. View Full-Text
Keywords: rare disease; orphan drugs; synthetic lethality; targeted cancer therapy; combination therapy; DNA repair; precision medicine; genomic instability; chemotherapy; clinical trials rare disease; orphan drugs; synthetic lethality; targeted cancer therapy; combination therapy; DNA repair; precision medicine; genomic instability; chemotherapy; clinical trials
Show Figures

Figure 1

MDPI and ACS Style

Bhattacharjee, S.; Nandi, S. Rare Genetic Diseases with Defects in DNA Repair: Opportunities and Challenges in Orphan Drug Development for Targeted Cancer Therapy. Cancers 2018, 10, 298. https://doi.org/10.3390/cancers10090298

AMA Style

Bhattacharjee S, Nandi S. Rare Genetic Diseases with Defects in DNA Repair: Opportunities and Challenges in Orphan Drug Development for Targeted Cancer Therapy. Cancers. 2018; 10(9):298. https://doi.org/10.3390/cancers10090298

Chicago/Turabian Style

Bhattacharjee, Sonali, and Saikat Nandi. 2018. "Rare Genetic Diseases with Defects in DNA Repair: Opportunities and Challenges in Orphan Drug Development for Targeted Cancer Therapy" Cancers 10, no. 9: 298. https://doi.org/10.3390/cancers10090298

Find Other Styles
Note that from the first issue of 2016, MDPI journals use article numbers instead of page numbers. See further details here.

Article Access Map by Country/Region

1
Back to TopTop