Disease: Acquired immunodeficiency syndrome HIV-1-infection

Disease info:


AIDS stands for acquired immunodeficiency syndrome. It is the final stage of infection with HIV. Not everyone with HIV develops AIDS.

It harms the immune system by destroying the white blood cells that fight infection. This puts you at risk for serious infections and certain cancers.

HIV most often spreads through unprotected sex with a person who has HIV. It may also spread by sharing drug needles or through contact with the blood of a person who has HIV. Women can give it to their babies during pregnancy or childbirth. 

The first signs of HIV infection may be swollen glands and flu-like symptoms. These may come and go within two to four weeks. Severe symptoms may not apurear until months or years later.

 

Frequency:
Approximately 1.1 million people in the U.S. are living with HIV today. About 14 percent of them (1 in 7) don’t know it and need testing.
Official title:
Safety and Feasibility Study of Allotransplantation of CRISPR/Cas9 CCR5 Gene Modified CD34+ Hematopoietic Stem/Progenitor Cells in HIV-infected Subjects With Hematological Malignances
Who:

Principal Investigator: Hu Chen, MD, PhD  Affiliated Hospital of Academy to Military Medical Sciences

Principal Investigator: Hongkui Deng, PhD  Affiliated Hospital of Academy to Military Medical Sciences       

Principal Investigator: Hao Wu, MD, PhD  Affiliated Hospital of Academy to Military Medical Sciences

Sponsor:

Affiliated Hospital to Academy of Military Medical Sciences

Locations:

China, Beijing

Study start:
May. 23, 2017
Enrollment:
5 participants
Gene editing method:
CRISPR-Cas9
Gene:
CCR5
Vector:
No information
Note:
Phase not applicable
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

The investigators performed this study to evaluate the safety and feasibility of transplantation with CRISPR/Cas9 CCR5 gene modified CD34+ hematopoietic stem/progenitor cells for patients that develop AIDS and hematological malignances. Patients will be treated with antiviral therapy (ART) to achieve undetectable HIV-1 virus in peripheral blood before conditioning. CD34+ cells from donors will be infused into the patients after treatment with CRISPR/Cas9 to ablate CCR5 gene.