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Supplement to Promote Diversity and Inclusion, Female Scientist

促进多样性和包容性的补充，女科学家

基本信息

批准号：
10057248
负责人：
Fernando Aleman Guillen
金额：
$ 9.51万
依托单位：
NAVEGA THERAPEUTICS, INC.
依托单位国家：
美国
项目类别：
财政年份：
2019
资助国家：
美国
起止时间：
2019-11-30 至 2020-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10057248
关键词：
Affect American Biological Assay CMV promoter Cells Congenital Pain Insensitivity Cytomegalovirus DNA DNA Sequence Databases Diabetes Mellitus Dideoxy Chain Termination DNA Sequencing Drug or chemical Tissue Distribution Enhancers Ensure Female Genes Genetic study Genome Guide RNA Heart Diseases Homologous Gene Human Human Cell Line In Vitro Individual Inherited Liposomes Malignant Neoplasms Mus Narcotics Nucleotides Opioid Orthologous Gene Pain Pain management Pathway interactions Patients Pattern Phase Phenotype Promoter Regions Property Protein Isoforms Proteins Quantitative Reverse Transcriptase PCR Reporter Repression Risk Safety Scientist Small Business Innovation Research Grant Sodium Channel Specificity Stretching Testing Therapeutic Toxic effect Toxicology Transcription Initiation Site Variant Viral Western Blotting Work Zinc Fingers addiction base chronic pain design exosome gene therapy genetic testing human DNA improved in silico in vitro testing in vivo indexing inflammatory neuropathic pain inhibitor/antagonist interest loss of function mutation mouse model non-opioid analgesic pain signal particle promoter rare genetic disorder side effect tool transcriptome sequencing transmission process vector voltage

项目摘要

Project Summary/Abstract Currently more than 100 million Americans and 1.5 billion people worldwide suffer from chronic pain. This is more than cancer, diabetes, and heart disease combined. Voltage-gated sodium channels are responsible for the transmission of pain signals. Nine genes have been identified, each having unique properties and tissue distribution patterns. Genetic studies have correlated a hereditary loss-of-function mutation in one human Na+ channel isoform – ?Na?V?1.7 – with a rare genetic disorder known as Congenital Insensitivity to Pain (CIP). Individuals with CIP are not able to feel pain without any significant secondary alteration. Thus, selective inhibition of ?Na?V?1.7 in normal humans could recapitulate the phenotype of CIP. However, the high homology of human ?Na?V proteins have frustrated most efforts to develop selective inhibitors. We have developed a non-permanent gene therapy to target pain that is non-addictive (because it targets a non-opioid pathway), highly specific (only targeting the gene of interest), and long-term lasting (around 3 weeks in preliminary assays in mice). During this Phase I SBIR, we will 1) test additional pain targets ?in vitro?, and 2) evaluate the new targets ?in vivo ?in mice models of inflammatory and neuropathic pain. In addition, we will initiate our toxicology studies in mice. At the end of this Phase I work, we will know the spectrum of activity and safety of our optimized candidate to perform IND-enabling toxicology studies in Phase II.

项目总结/文摘