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Engineering the next generation of cell-based therapeutics

期刊

NATURE REVIEWS DRUG DISCOVERY
卷 21, 期 9, 页码 655-675

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NATURE PORTFOLIO
DOI: 10.1038/s41573-022-00476-6

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资金

  1. JDRF [JDRF 3-SRA-2021-1023-S-B]
  2. US NIH [R01DK120459, R35GM143532, R21EB030772, R01EB032272]
  3. ONR [N00014-21-1-4006]
  4. Cancer Prevention & Research Institute of Texas (CPRIT) [RR170030, RP210205]

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The widespread clinical translation and commercialization of cell-based therapies are hindered by challenges related to cell source, product quality, and manufacturing processes. However, biological engineering approaches such as genome editing, synthetic biology, and the use of biomaterials are gradually addressing these issues.
The widespread clinical translation and commercialization of cell-based therapies are hampered by challenges related to cell source, viability, potency, safety and scalability. Here, Veiseh and colleagues overview progress in the development of cell-based therapeutics and discuss how biological engineering approaches - including genome editing, synthetic biology and the use of biomaterials - are beginning to address key challenges in the field. Cell-based therapeutics are an emerging modality with the potential to treat many currently intractable diseases through uniquely powerful modes of action. Despite notable recent clinical and commercial successes, cell-based therapies continue to face numerous challenges that limit their widespread translation and commercialization, including identification of the appropriate cell source, generation of a sufficiently viable, potent and safe product that meets patient- and disease-specific needs, and the development of scalable manufacturing processes. These hurdles are being addressed through the use of cutting-edge basic research driven by next-generation engineering approaches, including genome and epigenome editing, synthetic biology and the use of biomaterials.

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