PMID- 34127193
OWN - NLM
STAT- MEDLINE
DCOM- 20211125
LR  - 20211125
IS  - 1878-0814 (Electronic)
IS  - 1877-1173 (Linking)
VI  - 181
DP  - 2021
TI  - CRISPR-Cas9 for treating hereditary diseases.
PG  - 165-183
LID - S1877-1173(21)00028-4 [pii]
LID - 10.1016/bs.pmbts.2021.01.017 [doi]
AB  - This chapter analyzes to use of the genome editing tool to the treatment of 
      various genetic diseases. The genome editing method could be used to change the 
      DNA in cells or organisms to understand their physiological response. Therefore, 
      a key objective is to present general information about the use of the genome 
      editing tool in a pertinent way. An emerging genome editing technology like a 
      clustered regularly short palindromic repeats (CRISPR) is an extensively expended 
      in biological sciences. CRISPR and CRISPR-associated protein 9 (CRISPR-Cas9) 
      technique is being utilized to edit any DNA mutations associated with hereditary 
      diseases to study in cells (in vitro) and animals (in vivo). Interestingly, 
      CRISPR-Cas9 could be used to the investigation of treatments of various human 
      hereditary diseases such as hemophila, beta-thalassemia, cystic fibrosis, 
      Alzheimer's, Huntington's, Parkinson's, tyrosinemia, Duchnene muscular dystrophy, 
      Tay-Sachs, and fragile X syndrome disorders. Furthermore, CRISPR-Cas9 could also 
      be used in other diseases to the improvement of human health. Finally, this 
      chapter discuss current progress to treatment for hereditary diseases using 
      CRISPR-Cas9 technology and highlights associated challenges and future prospects.
CI  - Copyright (c) 2021 Elsevier Inc. All rights reserved.
FAU - Mani, Indra
AU  - Mani I
AD  - Department of Microbiology, Gargi College, University of Delhi, New Delhi, India. 
      Electronic address: indramanibhu@gmail.com.
LA  - eng
PT  - Journal Article
DEP - 20210224
PL  - Netherlands
TA  - Prog Mol Biol Transl Sci
JT  - Progress in molecular biology and translational science
JID - 101498165
SB  - IM
MH  - Animals
MH  - *CRISPR-Cas Systems/genetics
MH  - Gene Editing
MH  - *Genetic Therapy
MH  - Humans
MH  - Mutation
OTO - NOTNLM
OT  - CRISPR
OT  - Cas-9
OT  - Genetic diseases
OT  - Genome editing
OT  - Mutations
EDAT- 2021/06/16 06:00
MHDA- 2021/11/26 06:00
CRDT- 2021/06/15 05:59
PHST- 2021/06/15 05:59 [entrez]
PHST- 2021/06/16 06:00 [pubmed]
PHST- 2021/11/26 06:00 [medline]
AID - S1877-1173(21)00028-4 [pii]
AID - 10.1016/bs.pmbts.2021.01.017 [doi]
PST - ppublish
SO  - Prog Mol Biol Transl Sci. 2021;181:165-183. doi: 10.1016/bs.pmbts.2021.01.017. 
      Epub 2021 Feb 24.