PMID- 37440022
OWN - NLM
STAT- MEDLINE
DCOM- 20230717
LR  - 20230901
IS  - 0065-2598 (Print)
IS  - 0065-2598 (Linking)
VI  - 1415
DP  - 2023
TI  - Generation of an Avian Myeloblastosis Virus (AMV) Reverse Transcriptase Prime 
      Editor.
PG  - 109-114
LID - 10.1007/978-3-031-27681-1_17 [doi]
AB  - Prime editing (PE) is a novel, double-strand break (DSB)-independent gene editing 
      technology that represents an exciting avenue for the treatment of inherited 
      retinal diseases (IRDs). Given the extensive and heterogenous nature of the 280 
      genes associated with IRDs, genome editing has presented countless complications. 
      However, recent advances in genome editing technologies have identified PE to 
      have tremendous potential, with the capability to ameliorate small deletions and 
      insertions in addition to all twelve possible transition and transversion 
      mutations. The current PE system is based on the fusion of the Streptococcus 
      pyogenes Cas9 (SpCas9) nickase H840A mutant and an optimized Moloney murine 
      leukemia virus (MMLV) reverse-transcriptase (RT) in conjunction with a PE guide 
      RNA (pegRNA). In this study, we developed a prime editor based on the avian 
      myeloblastosis virus (AMV)-RT and showed its applicability for the installation 
      of the PRPH2 c.828+1G>A mutation in HEK293 cells.
CI  - (c) 2023. The Author(s), under exclusive license to Springer Nature Switzerland AG.
FAU - Tsai, Yi-Ting
AU  - Tsai YT
AD  - Department of Biomedical Engineering, Columbia University, New York, NY, USA.
FAU - da Costa, Bruna Lopes
AU  - da Costa BL
AD  - Department of Biomedical Engineering, Columbia University, New York, NY, USA.
AD  - Edward S. Harkness Eye Institute, Department of Ophthalmology, Columbia 
      University Irving Medical Center/New York-Presbyterian Hospital, New York, NY, 
      USA.
AD  - Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, 
      Department of Ophthalmology, Columbia University, New York, NY, USA.
FAU - Caruso, Salvatore Marco
AU  - Caruso SM
AD  - Department of Biomedical Engineering, Columbia University, New York, NY, USA.
AD  - Edward S. Harkness Eye Institute, Department of Ophthalmology, Columbia 
      University Irving Medical Center/New York-Presbyterian Hospital, New York, NY, 
      USA.
AD  - Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, 
      Department of Ophthalmology, Columbia University, New York, NY, USA.
AD  - Columbia Stem Cell Initiative, Columbia University, New York, NY, USA.
FAU - Nolan, Nicolas D
AU  - Nolan ND
AD  - Department of Biomedical Engineering, Columbia University, New York, NY, USA.
AD  - Edward S. Harkness Eye Institute, Department of Ophthalmology, Columbia 
      University Irving Medical Center/New York-Presbyterian Hospital, New York, NY, 
      USA.
AD  - Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, 
      Department of Ophthalmology, Columbia University, New York, NY, USA.
AD  - Columbia Stem Cell Initiative, Columbia University, New York, NY, USA.
FAU - Levi, Sarah R
AU  - Levi SR
AD  - Edward S. Harkness Eye Institute, Department of Ophthalmology, Columbia 
      University Irving Medical Center/New York-Presbyterian Hospital, New York, NY, 
      USA.
AD  - Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, 
      Department of Ophthalmology, Columbia University, New York, NY, USA.
FAU - Tsang, Stephen H
AU  - Tsang SH
AD  - Department of Biomedical Engineering, Columbia University, New York, NY, USA.
AD  - Edward S. Harkness Eye Institute, Department of Ophthalmology, Columbia 
      University Irving Medical Center/New York-Presbyterian Hospital, New York, NY, 
      USA.
AD  - Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, 
      Department of Ophthalmology, Columbia University, New York, NY, USA.
AD  - Columbia Stem Cell Initiative, Columbia University, New York, NY, USA.
AD  - Department of Pathology & Cell Biology, Columbia University, New York, NY, USA.
AD  - Institute of Human Nutrition, Columbia University, New York, NY, USA.
FAU - Quinn, Peter M J
AU  - Quinn PMJ
AD  - Edward S. Harkness Eye Institute, Department of Ophthalmology, Columbia 
      University Irving Medical Center/New York-Presbyterian Hospital, New York, NY, 
      USA. pq2138@cumc.columbia.edu.
AD  - Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, 
      Department of Ophthalmology, Columbia University, New York, NY, USA. 
      pq2138@cumc.columbia.edu.
LA  - eng
PT  - Journal Article
PL  - United States
TA  - Adv Exp Med Biol
JT  - Advances in experimental medicine and biology
JID - 0121103
RN  - EC 2.7.7.49 (RNA-Directed DNA Polymerase)
SB  - IM
MH  - Humans
MH  - Animals
MH  - Mice
MH  - *RNA-Directed DNA Polymerase/genetics/metabolism
MH  - *Avian Myeloblastosis Virus/genetics/metabolism
MH  - HEK293 Cells
MH  - Gene Editing
MH  - Moloney murine leukemia virus/genetics/metabolism
MH  - CRISPR-Cas Systems
OTO - NOTNLM
OT  - AMV-RT prime editors
OT  - Avian myeloblastosis virus (AMV)
OT  - PRPH2
OT  - Prime editing
OT  - Retinitis pigmentosa (RP)
EDAT- 2023/07/13 12:31
MHDA- 2023/07/17 06:42
CRDT- 2023/07/13 11:15
PHST- 2023/07/17 06:42 [medline]
PHST- 2023/07/13 12:31 [pubmed]
PHST- 2023/07/13 11:15 [entrez]
AID - 10.1007/978-3-031-27681-1_17 [doi]
PST - ppublish
SO  - Adv Exp Med Biol. 2023;1415:109-114. doi: 10.1007/978-3-031-27681-1_17.