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Insights of CRISPR-Cas systems in stem cells: progress in regenerative medicine

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Abstract

Regenerative medicine, a therapeutic approach using stem cells, aims to rejuvenate and restore the normalized function of the cells, tissues, and organs that are injured, malfunctioning, and afflicted. This influential technology reaches its zenith when it is integrated with the CRISPR-Cas (clustered regularly interspaced short palindromic repeats—CRISPR associated) technology of genome editing. This tool acts as a programmable restriction enzyme system, which targets DNA as well as RNA and gets redeployed for the customization of DNA/RNA sequences. The dynamic behaviour of nuclear manipulation and transcriptional regulation by CRISPR-Cas technology renders it with numerous employment in the field of biologics and research. Here, the possible impact of the commonly practiced CRISPR-Cas systems in regenerative medicines is being reviewed. Primarily, the discussion of the working mechanism of this system and the fate of stem cells will be scrutinized. A detailed description of the CRISPR based regenerative therapeutic approaches for a horde of diseases like genetic disorders, neural diseases, and blood-related diseases is elucidated.

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Abbreviations

CRISPR-Cas:

Clustered repeat interspaced short palindromic repeats/CRISPR associated proteins

iPSC:

Induced pluripotent stem cells

TALEN:

Transcription activator-like endonucleases

ZFN:

Zinc finger nucleases

RNP:

Ribonucleotide protein

PAM:

Protospacer adjacent motiff

crRNA:

CRISPR RNA

NHEJ:

Non homologous end joining

HDR:

Homology-directed repair

tracrRNA:

Trans activating crRNA

dsRNA:

Double stranded RNA

BMP4:

Bone morphogenic protein 4

UM-MSC:

Umbilical cord-derived mesenchymal stem cells

OM:

Osteogenic differentiation media

CRISPRai:

CRISPR activation/repression

BMSC:

Bone marrow-derived mesenchymal stem cells

GDNF:

Glial cell line-derived growth factor

BDNF:

Brain-derived neurotrophic factor

NGF:

Nerve growth factor

ALS:

Amyotrophic lateral sclerosis

sALS:

Sporadic amyotrophic lateral sclerosis

fALS:

Familial amyotrophic lateral sclerosis

SOD1:

Cu–Zn superoxide dismutase 1

TDP-43:

TAR DNA binding protein 43

VCP:

Valosin containing protein

ANG:

Angiogenin

OPTN:

Optineurin

HLC:

Hepatocyte-like cells

RDEB:

Recessive dystrophic epidermolysisbullosa

DMD:

Duchenne muscular dystrophy

OA:

Osteoarthritis

PH1:

Primary hyperoxaluria type 1

CFTR:

Cystic fibrosis transmembrane regulator

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Acknowledgements

Authors are thankful to The Management of Sri Venkateswara College of Engineering, Sriperumbudur, Chennai, Tamil Nadu, India, Management of Sree Sastha Institute of Engineering and Technology, Chembarambakkam, Chennai, Tamil Nadu, India and The Management, Vice Chancellor, Dean of SMNS and Head of Biological Sciences, The Copperbelt University, Kitwe, Zambia for their constant support to complete the review article.

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Authors and Affiliations

  1. Department of Biotechnology, Sri Venkateswara College of Engineering, Sriperumbudur, Chennai, Tamil Nadu, 602 117, India

    Shanmugam Dilip Kumar & Guruviah KarthigaDevi

  2. Department of Biotechnology, Sree Sastha Institute of Engineering and Technology, Chembarambakkam, Chennai, Tamil Nadu, 600 123, India

    Manimaran Aashabharathi

  3. Department of Biological Sciences, School of Mathematics and Natural Sciences, The Copperbelt University, Riverside, Jambo Drive, P.O Box. 21692, Kitwe, Zambia

    Ramasamy Subbaiya

  4. AMR and Nanomedicine Laboratory, Department of Pharmacology, Saveetha Dental College, Saveetha Institute of Medical and Technical Sciences (SIMATS), Chennai, Tamil Nadu, 600 077, India

    Muthupandian Saravanan

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  1. Shanmugam Dilip Kumar
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  2. Manimaran Aashabharathi
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  3. Guruviah KarthigaDevi
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SDK, MA and GK: literature collection, writing—original draft, Methodology, Resources. RS and MS: conceptualization, supervision, validation, writing—review & editing for the final version manuscript.

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Dilip Kumar, S., Aashabharathi, M., KarthigaDevi, G. et al. Insights of CRISPR-Cas systems in stem cells: progress in regenerative medicine. Mol Biol Rep 49, 657–673 (2022). https://doi.org/10.1007/s11033-021-06832-w

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