相关实验视频

Updated: Jun 21, 2026

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
11:16

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

8.0K

在体内对人类造血干细胞和祖细胞进行基因编辑,使用外工程病毒样颗粒进行编辑

Vladimir V Botchkarev1, Sean Harrington1, Matteo Stoppato1

  • 1Sana Biotechnology, Inc., Cambridge, MA, USA.

Nature biotechnology
|December 5, 2025
PubMed
概括

工程病毒样颗粒 (VLPs) 提供了一种新的方法来编辑人类干细胞中的基因. 这些VLP通过精确向造血干细胞和原始细胞 (HSPCs) 来治疗遗传性血液疾病具有前途.

更多相关视频

Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models08:14

Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models

Published on: October 3, 2019

12.9K
CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications08:32

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

Published on: August 9, 2022

4.2K

相关概念视频

What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview
In-vitro Mutagenesis01:16

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
CRISPR01:59

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced Short...
CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...