Disease: Human Papillomavirus-Related Malignant Neoplasm

Disease info:

Human Papillomavirus-HPV persistent infection is the major causal factor of cervical intraepithelial neoplasia (CIN) and cervical cancer.

Two HPV types (16 and 18) cause 70% of cervical cancers and pre-cancerous cervical lesions.

Frequency:
Globally, 12% of women are positive for HPV DNA, with rates varying by age and country. The highest rates of HPV are in younger women, with a rate of 24% in women under 25 years.
Official title:
A Safety and Efficacy Study of Transcription Activator-like Effector Nucleases and Clustered Regularly Interspaced Short Palindromic Repeat/Cas9 in the Treatment of HPV-related Cervical Intraepithelial NeoplasiaⅠ
Who:

Principal Investigator: Zheng Hu, M.D.  First Affiliated Hospital, Sun Yat-Sen University
 

Locations:

China, Guangdong

 

Study start:
Jan. 15, 2018
Enrollment:
60 participants
Gene editing method:
CRISPR-Cas9 and TALEN
Gene:
HPV16 and HPV18 E6/E7
Vector:
No information
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Unknown

Description

This is an open-label and triple cohort study of the safety and efficacy of TALEN and CRISPR-Cas9 to possibly treat HPV Persistency and human cervical intraepithelial neoplasia Ⅰ without invasion.

HPV persistent infection is the major causal factor of cervical intraepithelial neoplasia (CIN) and cervical cancer.
The important roles of E6 and E7 playing in HPV-driven carcinogenesis make them attractive targets for therapeutic interventions. Previous evidences showed that using designated TALEN and CRISPR-Cas9 as genome editing tool could produce disruption of HPV16 and HPV18 E6/E7 DNA, significantly decreasing the expression of E6/E7, inducing cell apoptosis and inhibiting cell lines growth.
TALEN or CRISPR plasmid administered in gel twice one week for 4 weeks.