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Related Experiment Video

Updated: Jul 26, 2026

Targeted Next-generation Sequencing and Bioinformatics Pipeline to Evaluate Genetic Determinants of Constitutional Disease

Targeted Next-generation Sequencing and Bioinformatics Pipeline to Evaluate Genetic Determinants of Constitutional Disease

Published on: April 4, 2018

The development of genomics-derived pharmaceuticals.

T M Beardsley¹, C A Rosen, D C Stump

¹Human Genome Sciences Inc, 9410 Key West Avenue, Rockville, MD 20850, USA. Timothy_Beardsley@hgsi.com

Current Opinion in Drug Discovery & Development

|May 31, 2001

Summary

Researchers have sequenced thousands of human genes involved in cell signaling, producing functional proteins. One protein has shown promising clinical activity in early trials, advancing therapeutic development.

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Area of Science:

Molecular Biology
Genomics
Biochemistry

Background:

Availability of messenger RNA (mRNA) sequences for most human genes.
Human Genome Sciences database contains mRNA sequences capable of producing functional proteins.
Cell signaling pathways are crucial for cellular function and disease.

Purpose of the Study:

To sequence approximately 8000 human gene clones involved in cell signaling.
To produce corresponding functional proteins from these clones.
To screen these proteins for biological activity and therapeutic potential.

Main Methods:

Sequencing of approximately 8000 human gene clones.
Production of small quantities of corresponding proteins.
Screening of proteins for biological activity.
Clinical trials for promising candidates.

Main Results:

Successful sequencing and protein production for a significant number of cell signaling genes.
Four candidate proteins have advanced to clinical trials.
One protein has demonstrated measurable clinical activity.

Conclusions:

The study successfully generated a library of cell signaling proteins.
The identified protein shows therapeutic promise, warranting further investigation.
This work contributes to the development of novel therapeutics targeting cell signaling pathways.