PMID- 33788143
OWN - NLM
STAT- MEDLINE
DCOM- 20220307
LR  - 20220307
IS  - 2629-3277 (Electronic)
IS  - 2629-3277 (Linking)
VI  - 17
IP  - 5
DP  - 2021 Oct
TI  - The Strategies and Challenges of CCR5 Gene Editing in Hematopoietic Stem and 
      Progenitor Cells for the Treatment of HIV.
PG  - 1607-1618
LID - 10.1007/s12015-021-10145-7 [doi]
AB  - HIV infection continues to be a serious health issue with an alarming global 
      spread, owing to the fact that attempts at developing an effective vaccine or a 
      permanent cure remains futile. So far, the only available treatment for the 
      clinical management of HIV is the combined Anti-Retroviral Therapy (cART), but 
      the long-term cART is associated with metabolic changes, organ damages, and 
      development and transmission of drug resistant HIV strains. Thus, there is a need 
      for the development of one-time curative treatment for HIV infection. The 
      allogeneic transplantation with the Hematopoietic Stem and Progenitor cells 
      (HSPCs) having 32 bp deletion in Chemokine receptor 5 gene (CCR5 Delta32) 
      demonstrated successful HIV remission in the Berlin and London patients, and 
      highlighted that transplantation of CCR5 null HSPCs is a promising approach for a 
      long- term HIV remission. The advent of gene editing technologies offers a new 
      choice of generating ex vivo CCR5 ablated allogeneic or autologous HSPCs for stem 
      cell transplantation into HIV patients. Many groups are attempting CCR5 
      disruption in HSPCs using various gene-editing strategies. At least two such 
      studies, involving CCR5 gene editing in HSPCs have entered the clinical trials. 
      This review aims to outline the strategies taken for CCR5 gene editing and 
      discuss the challenges associated with the development of CCR5 manipulated HSPCs 
      for the gene therapy of HIV infection.
CI  - (c) 2021. The Author(s), under exclusive licence to Springer Science+Business 
      Media, LLC, part of Springer Nature.
FAU - Karuppusamy, Karthik V
AU  - Karuppusamy KV
AD  - Centre for Stem Cell Research (A unit of inStem, Bengaluru), Christian Medical 
      College, Vellore, Tamil Nadu, India.
AD  - Manipal Academy of Higher Education, Manipal, Karnataka, India.
FAU - Babu, Prathibha
AU  - Babu P
AD  - Centre for Stem Cell Research (A unit of inStem, Bengaluru), Christian Medical 
      College, Vellore, Tamil Nadu, India.
AD  - Manipal Academy of Higher Education, Manipal, Karnataka, India.
FAU - Thangavel, Saravanabhavan
AU  - Thangavel S
AUID- ORCID: 0000-0001-6760-4106
AD  - Centre for Stem Cell Research (A unit of inStem, Bengaluru), Christian Medical 
      College, Vellore, Tamil Nadu, India. sthangavel@cmcvellore.ac.in.
AD  - Manipal Academy of Higher Education, Manipal, Karnataka, India. 
      sthangavel@cmcvellore.ac.in.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Review
DEP - 20210331
PL  - United States
TA  - Stem Cell Rev Rep
JT  - Stem cell reviews and reports
JID - 101752767
RN  - 0 (CCR5 protein, human)
RN  - 0 (Receptors, CCR5)
SB  - IM
MH  - Gene Editing
MH  - *HIV Infections/genetics/therapy
MH  - *Hematopoietic Stem Cell Transplantation
MH  - Humans
MH  - Receptors, CCR5/genetics
OTO - NOTNLM
OT  - CCR5
OT  - Gene editing
OT  - HIV gene therapy
OT  - Hematopoietic stem cells
EDAT- 2021/04/01 06:00
MHDA- 2022/03/08 06:00
CRDT- 2021/03/31 13:29
PHST- 2021/02/24 00:00 [accepted]
PHST- 2021/04/01 06:00 [pubmed]
PHST- 2022/03/08 06:00 [medline]
PHST- 2021/03/31 13:29 [entrez]
AID - 10.1007/s12015-021-10145-7 [pii]
AID - 10.1007/s12015-021-10145-7 [doi]
PST - ppublish
SO  - Stem Cell Rev Rep. 2021 Oct;17(5):1607-1618. doi: 10.1007/s12015-021-10145-7. 
      Epub 2021 Mar 31.