Genome Editing with Crispr-Cas9 Systems: Basic Research and Clinical Applications
BACKGROUND: Recently established genome editing technologies will open new avenues for biological research and development. Human genome editing is a powerful tool which offers great scientific and therapeutic potential. CONTENT: Genome editing using the clustered regularly interspaced short palind...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
Secretariat of The Indonesian Biomedical Journal
2017-04-01
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| Series: | Indonesian Biomedical Journal |
| Online Access: | https://inabj.org/index.php/ibj/article/view/272 |
| _version_ | 1811213248361398272 |
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| author | Anna Meiliana Nurrani Mustika Dewi Andi Wijaya |
| author_facet | Anna Meiliana Nurrani Mustika Dewi Andi Wijaya |
| author_sort | Anna Meiliana |
| collection | DOAJ |
| description | BACKGROUND: Recently established genome editing technologies will open new avenues for biological research and development. Human genome editing is a powerful tool which offers great scientific and therapeutic potential.
CONTENT: Genome editing using the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPRassociated protein 9 (Cas9) technology is revolutionizing the gene function studies and possibly will give rise to an entirely new degree of therapeutics for a large range of diseases. Prompt advances in the CRISPR/Cas9 technology, as well as delivery modalities for gene therapy applications, are dismissing the barriers to the clinical translation of this technology. Many studies conducted showed promising results, but as current available technologies for evaluating off-target gene modification, several elements must be addressed to validate the safety of the CRISPR/Cas9 platform for clinical application, as the ethical implication as well.
SUMMARY: The CRISPR/Cas9 system is a powerful genome editing technology with the potential to create a variety of novel therapeutics for a range of diseases, many of which are currently untreatable.
KEYWORDS: genome editing, CRISPR-Cas, guideRNA, DSB, ZFNs, TALEN |
| first_indexed | 2024-04-12T05:42:43Z |
| format | Article |
| id | doaj.art-05229fd816424e80bea21d5734860039 |
| institution | Directory Open Access Journal |
| issn | 2085-3297 2355-9179 |
| language | English |
| last_indexed | 2024-04-12T05:42:43Z |
| publishDate | 2017-04-01 |
| publisher | Secretariat of The Indonesian Biomedical Journal |
| record_format | Article |
| series | Indonesian Biomedical Journal |
| spelling | doaj.art-05229fd816424e80bea21d57348600392022-12-22T03:45:33ZengSecretariat of The Indonesian Biomedical JournalIndonesian Biomedical Journal2085-32972355-91792017-04-019111610.18585/inabj.v9i1.272230Genome Editing with Crispr-Cas9 Systems: Basic Research and Clinical ApplicationsAnna Meiliana0Nurrani Mustika Dewi1Andi Wijaya2Postgraduate Program in Clinical Pharmacy, Padjadjaran UniversityPostgraduate Program in Clinical Pharmacy, Padjadjaran UniversityProdia Clinical Laboratory, Jl. Cisangkuy No. 2, Bandung 40114BACKGROUND: Recently established genome editing technologies will open new avenues for biological research and development. Human genome editing is a powerful tool which offers great scientific and therapeutic potential. CONTENT: Genome editing using the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPRassociated protein 9 (Cas9) technology is revolutionizing the gene function studies and possibly will give rise to an entirely new degree of therapeutics for a large range of diseases. Prompt advances in the CRISPR/Cas9 technology, as well as delivery modalities for gene therapy applications, are dismissing the barriers to the clinical translation of this technology. Many studies conducted showed promising results, but as current available technologies for evaluating off-target gene modification, several elements must be addressed to validate the safety of the CRISPR/Cas9 platform for clinical application, as the ethical implication as well. SUMMARY: The CRISPR/Cas9 system is a powerful genome editing technology with the potential to create a variety of novel therapeutics for a range of diseases, many of which are currently untreatable. KEYWORDS: genome editing, CRISPR-Cas, guideRNA, DSB, ZFNs, TALENhttps://inabj.org/index.php/ibj/article/view/272 |
| spellingShingle | Anna Meiliana Nurrani Mustika Dewi Andi Wijaya Genome Editing with Crispr-Cas9 Systems: Basic Research and Clinical Applications Indonesian Biomedical Journal |
| title | Genome Editing with Crispr-Cas9 Systems: Basic Research and Clinical Applications |
| title_full | Genome Editing with Crispr-Cas9 Systems: Basic Research and Clinical Applications |
| title_fullStr | Genome Editing with Crispr-Cas9 Systems: Basic Research and Clinical Applications |
| title_full_unstemmed | Genome Editing with Crispr-Cas9 Systems: Basic Research and Clinical Applications |
| title_short | Genome Editing with Crispr-Cas9 Systems: Basic Research and Clinical Applications |
| title_sort | genome editing with crispr cas9 systems basic research and clinical applications |
| url | https://inabj.org/index.php/ibj/article/view/272 |
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