関連する実験動画

Updated: Sep 9, 2025

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

7.8K

レンチウイルスベクターと血液形成幹細胞による遺伝子治療のリスクと利益の評価: スキソナ事件

Pilar Puig-Serra1, Ana Hinckley-Boned2,3, María Tristán-Manzano2,3,4

  • 1Molecular Cytogenetics and Genome Editing Unit, Human Cancer Genetics Program, Centro Nacional de Investigaciones Oncológicas (CNIO), Madrid, Spain.

Human gene therapy
|September 1, 2025
PubMed
まとめ

スキソナTM (elivaldogene autotemcel) がんによるレンチウイルスベクター遺伝子療法の安全性に関する懸念が再び浮上しました. しかし,他のレンチウイルスベクトル製品は,リスクをさらに軽減するために進行中の研究で,好ましい安全性プロファイルを示しています.

キーワード:
スキソナ遺伝子療法の安全性遺伝子毒性造血幹細胞挿入型変異レンチウイルスベクター

さらに関連する動画

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

3.7K
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

12.4K

関連する概念動画

Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
25.8K
Bone Marrow Sampling and Transplants01:22

Bone Marrow Sampling and Transplants

Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
493
Embryonic Stem Cells00:57

Embryonic Stem Cells

Embryonic stem (ES) cells were first discovered in mice in 1981 by Martin Evans. In 1998, James Thomson identified a method to isolate embryonic stem cells from humans. Human embryonic stem cells (hESCs) are obtained from 3-5 day old embryos that remain unused after an in vitro fertilization procedure.
ES cells are grown in a culture medium where they can divide indefinitely, creating ES cell lines. Under certain conditions, ES cells can differentiate, either spontaneously into a variety of...
3.7K