Difference Between Ex Vivo and In Vivo Gene Therapy

关键区别 - 体内基因治疗

基因治疗是一种重要的技术，用于通过引入缺失或缺陷基因的基因来治疗或预防遗传疾病。可以通过插入健康的基因代替导致该疾病的突变基因来治愈某些疾病。基因疗法主要用于体细胞比生殖细胞，可以分为两种称为Ex Vivo基因疗法和体内基因治疗的主要类型。离体和体内基因疗法之间的关键区别在于therapeutic genes are transferred to体外cell cultures and reintroduced into a patient in ex vivo gene therapywhile基因直接传递到患者的组织或细胞，而无需培养细胞体外在体内基因治疗。

内容
1.Overview and Key Difference
2.What is Ex Vivo Gene Therapy
3.体内其他基因是什么apy
4.Side by Side Comparison – Ex Vivo vs In Vivo Gene Therapy
5.概括

What is Ex Vivo Gene Therapy?

Ex vivo gene therapy is a type of gene therapy which involves exterior modification of a patient’s cell and reintroduction of it to the patient. The cells are cultured in the labs (outside the patient’s body), and genes are inserted. Then the stabletransformantsare selected and reintroduced into the patient to treat the disease. Ex vivo gene therapy can be applied only to certain cell types or selected tissues. Bone marrow cells are the cells frequently used for ex vivo gene therapy.

Procedure of Ex Vivo Gene Therapy

在体内基因治疗中涉及几个主要步骤，如下所示。

1. 从患者中分离出有缺陷基因的细胞。
2. 分离的细胞在实验室的培养物中生长。
3. 使用载体将治疗基因插入或介绍给种植的细胞培养物。
4. Transformed cells are selected from the nontransformants and grown.
5. Selected cells are transplanted into the patient.

在离体基因疗法中，carriers or vectors用于将基因输送到靶细胞中。成功的基因递送取决于载体系统，并且在体内基因治疗中使用的重要矢量是viruses, bone marrow cells, human artificial chromosome, etc. Compared to the in vivo gene therapy, ex vivo gene therapy does not involve adverse immunological reactions in the patient’s body since the genetic correction is done体外。但是，成功取决于稳定的补救基因在患者体内的表达和表达。

什么是体内基因疗法？

In vivo gene therapy is a technique which involves direct delivery of genes into the cells of a particular tissue inside the patient’s body to treat genetic diseases. It can be applied to many tissues of the human body including liver, muscle, skin, lung, spleen, brain, blood cells, etc. The therapeutic genes are introduced by the viral or nonviral-based vectors into the patient. However, the success depends on several factors such as efficient uptake of the therapeutic gene carrying vectors by the target cells, intracellular degradation of the genes within the target cells and gene uptake by the nucleus, expression ability of the gene, etc.

What is the difference between Ex Vivo and In Vivo Gene Therapy?

体内体内基因治疗
Ex vivo gene therapy is a type of gene therapy which is performed exterior to the patient’s body. Gene modification is done outside the body.	体内基因疗法是另一种基因疗法，当缺陷细胞仍在体内时直接进行。当基因仍在体内时，它们会改变。
Isolation and Culturing
有缺陷的细胞在实验室中分离并培养。	Defective cells are not isolated or cultured in the lab.
选择转化体
在重新引入之前选择稳定的转化体。	Stable transformants cannot be selected.
Adverse Immunological Response
This method does not introduce adverse immunological responses in the patient’s body.	This method introduces adverse immunological responses in the patient’s body.

摘要 - 体内基因治疗

Therapeutic genes are introduced into patient’s body as a treatment for certain diseases. It is known as gene therapy and can be done in two ways namely ex vivo gene therapy and in vivo gene therapy. The difference between ex vivo and in vivo gene therapy is that gene insertion in ex vivo gene therapy is done in the cell cultures exterior to patient’s body and the corrected cells are reintroduced to the patient while in in vivo gene therapy genes are introduced directly into the interior target tissues without isolating the cells. The success of the both processes depends on the stable insertion and transformation of the therapeutic genes into the patient cells.

Reference:
1.”什么是基因疗法？- 遗传学家庭参考。”美国国家医学图书馆。美国国立卫生研究院网络。2017年4月24日。
2.”Evaluation of the Clinical Success of Ex Vivo and In Vivo Gene Therapy | JYI – The Undergraduate Research Journal.” JYI The Undergraduate Research Journal. N.p., n.d. Web. 24 Apr. 2017
3. Crystal, Ronald G. “In vivo and ex vivo gene therapy strategies to treat tumors using adenovirus gene transfer vectors.” SpringerLink. Springer-Verlag, n.d. Web. 24 Apr. 2017

Image Courtesy:
1. “ExVivoGeneTherapy pl”By Pisum na podstawie pracy Lizanne Koch – Own work(CC BY-SA 3.0)viaCommons Wikimedia
2. “In vivo gene therapy pl” By Pisum na podstawie pracy Lizanne Koch – Own work(CC BY-SA 3.0)viaCommons Wikimedia