Overview of disease pathways and drug targets

Biomarkers, Critical Disease Pathways, Drug Targets, and Alternative Medicine in Male Breast Cancer

Current Drug Targets ◽

10.2174/138945009787122842 ◽

2009 ◽

Vol 10 (1) ◽

pp. 1-8 ◽

Cited By ~ 6

Author(s):

Debmalya Barh

Keyword(s):

Breast Cancer ◽

Alternative Medicine ◽

Male Breast Cancer ◽

Drug Targets ◽

Male Breast ◽

Disease Pathways

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Disease Pathways and Mechanisms of Potential Drug Targets

Clinical Liver Disease ◽

10.1002/cld.735 ◽

2018 ◽

Vol 12 (1) ◽

pp. 12-18 ◽

Cited By ~ 1

Author(s):

Marc G. Ghany ◽

Timothy M. Block

Keyword(s):

Drug Targets ◽

Potential Drug ◽

Potential Drug Targets ◽

Disease Pathways

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Gene Knock Up via 3’UTR editing to study gene function in vivo

10.1101/775031 ◽

2019 ◽

Author(s):

Kärt Mätlik ◽

Soophie Olfat ◽

Daniel R. Garton ◽

Ana Montaño-Rodriguez ◽

Giorgio Turconi ◽

...

Keyword(s):

Drug Targets ◽

Wild Type Mouse ◽

Copy Number Variations ◽

Endogenous Gene ◽

Entry Points ◽

Unknown Gene ◽

Cell Type Specific ◽

Specific Regulation ◽

Disease Pathways

AbstractCurrently available genetic tools do not allow researchers to upregulate (‘Knock Up’) the levels of a given protein while retaining its cell-type-specific regulation. As a result, we have limited ability to develop overexpression-related disease models, to study the contribution of single genes in diseases caused by copy number variations and to identify disease pathways for drug targets. Here we develop two approaches for endogenous gene upregulation: conditional Knock Up (cKU) utilizing the Cre/lox system, and CRISPR-Cas9 mediated gene Knock Up (KU) in wild-type mouse embryos and human cells. Using glial cell line derived neurotrophic factor (GDNF) as a proof of concept, we show that both approaches resulted in upregulation of endogenous GDNF levels without disturbing Gdnf’s expression pattern. Furthermore, CNS-specific GDNF cKU resulted in dopaminergic abnormalities and schizophrenia-like phenotypes. Our results suggest that gene Knock Up can reveal unknown gene functions and provide novel entry points for studying neurological disease.

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