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  1. 10 Απρ 2017 · Therapeutic gene editing can be administered through two basic strategies: (1) direct in vivo delivery of a gene-editing nuclease and (2) delivery of cells engineered ex vivo to contain a...

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  2. 18 Δεκ 2020 · Gene-editing technology is an emerging therapeutic modality for manipulating the eukaryotic genome by using target-sequence-specific engineered nucleases.

  3. 30 Απρ 2024 · The development of new PE variants holds significant promise for improving therapeutic gene editing by increasing editing efficiency, expanding targeting range, reducing off-target effects, multiplexing editing capability, enhancing delivery systems, and reducing immunogenicity.

  4. 1 Ιουλ 2024 · The purpose of this paper is three-fold: first, a chronological description of the history of CRISPR-Cas9-sgRNA-based gene editing; second, a brief description of the current state of clinical research in hematologic diseases, including selected applications in treating hematologic diseases with CRISPR-based gene therapy, preceded by a brief ...

  5. 17 Αυγ 2021 · Over the years biological therapy evolved from using stem cells and viral vectors to RNA therapy and testing different genome editing tools as promising gene therapy agents.

  6. 2 Δεκ 2019 · Abstract. Eukaryotic cells deploy overlapping repair pathways to resolve DNA damage. Advancements in genome editing take advantage of these pathways to produce permanent genetic changes....

  7. 1 Ιαν 2019 · Gene editing techniques have benefits such as: the treatment of diseases; creation of model organisms for basic biomedical research; development of transgenic foods, among other applications.

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