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  • 基因治疗

    Hands manipulating a DNA double helix with tools, symbolizing gene therapy techniques.

    Can doctors fix your ?
    ::医生能治好你吗?

    There are many that are due to a single gene . What if we could fix this faulty gene? With the development of gene therapy, that may eventually be possible for many types of genetic disorders.
    ::许多基因都是由单一基因造成的。如果我们能修复这个有缺陷的基因,那又如何?随着基因疗法的发展,这最终可能适用于许多类型的基因疾病。

    Gene Therapy
    ::基因治疗

    Gene therapy is the insertion of genes into a person’s cells to cure a genetic disorder. Could gene therapy be the cure for AIDS? No, AIDS is caused by a . Gene therapy only works to fix disorders caused by a faulty gene. The patient would have had this disorder from birth. Though gene therapy is still in experimental stages, the common use of this therapy may occur during your lifetime.
    ::基因疗法是将基因纳入人体细胞以治愈基因紊乱。基因疗法能否治愈艾滋病? 不,艾滋病是由一个 。 基因疗法只能修复由缺陷基因引起的病症。 病人从出生起就会患这种病。 尽管基因疗法仍处于实验阶段,但这种疗法的常用可能发生在你的一生中。

    There are two main types of gene therapy:
    ::基因疗法有两大类:

    1. One done inside the body ( in vivo ).
      ::一个在身体内(活体内)完成。
    2. One done outside the body ( ex vivo ).
      ::一个在身体外完成(体外)。

    Both types of gene therapy use a vector , or carrier molecule for the gene. The vector helps incorporate the desired gene into the patient's DNA. Usually this vector is modified viral DNA in which the viral genes have been removed. Don't worry, the virus used in gene therapy has been deactivated.
    ::这两种基因疗法都使用矢量或载体分子进行基因治疗。 矢量有助于将所需的基因纳入病人的DNA中。 通常, 该矢量是经过修改的病毒DNA, 病毒基因已被移除。 别担心, 基因疗法中使用的病毒已被停止使用 。

    In Vivo Gene Therapy
    ::维沃基因治疗

    During in vivo gene therapy, done inside the body, the vector with the gene of interest is introduced directly into the patient and taken up by the patient’s cells ( Figure ). For example, cystic fibrosis gene therapy is targeted at the , so a solution with the vector can be sprayed into the patient’s nose. Recently, in vivo gene therapy was also used to partially restore the of three young adults with a rare type of eye disease.
    ::在体内进行体外基因治疗期间,带有有关基因的病媒直接引入病人,并被病人的细胞接受(图 ) 。 比如,细胞纤维化基因疗法针对病人的细胞,因此病媒的溶液可以喷洒到病人的鼻子。 最近,在体外基因疗法中也使用了部分恢复3名患有罕见眼病的年轻人。

    Adenovirus vector delivering new gene into a cell during gene therapy process.

    During gene therapy, adenovirus is a possible vector to carry the desired gene and insert it into the patient's DNA. A deactivated virus makes a useful vector for this purpose.
    ::在基因治疗期间,肾脏病毒是一种可能携带所需基因并插入病人DNA的媒介。

    Ex Vivo Gene Therapy
    ::Ex Vivo Gene 治疗

    In ex vivo gene therapy, done outside the body, cells are removed from the patient and the proper gene is inserted using a virus as a vector. The modified cells are placed back into the patient.
    ::在人体外进行的异活基因治疗中,细胞从病人身上取出,并用病毒作为媒介插入适当的基因。修改后的细胞被放回病人体内。

    One of the first uses of this type of gene therapy was in the treatment of a young girl with a rare genetic disease, adenosine deaminase deficiency, or ADA deficiency. People with this disorder are missing the ADA enzyme , which breaks down a toxin called deoxyadenosine. If the toxin is not broken down, it accumulates and destroys immune cells. As a result, individuals with ADA deficiency do not have a healthy immune system to fight off infections. In the gene therapy treatment for this disorder, bone marrow stem cells were taken from the girl’s body, and the missing gene was inserted into these cells outside the body. Then the modified cells were put back into her bloodstream. This treatment successfully restored the function of her immune system, but only with repeated treatments.
    ::这种基因疗法的首批用途之一是治疗患有罕见遗传疾病、肾上腺素脱氧素缺乏症或ADA缺乏症的年轻女孩。患有这种疾病的人缺少ADA酶,这种酶会分解一种叫做脱氧乙酸的毒素。如果毒素没有分解,它会积累并摧毁免疫细胞。因此,患有ADA缺乏症的人没有健康的免疫系统来对抗感染。在治疗这种疾病的基因疗法中,从女孩的身体中取出了骨髓干干细胞,而失踪的基因被插入到身体外的这些细胞中。随后,改型细胞又被放回她的血液中。这种治疗成功地恢复了她的免疫系统的功能,但只是反复治疗。

    Summary
    ::摘要

    • Gene therapy, the insertion of genes into a person’s cells to cure a genetic disorder, can be ex vivo (outside the body) or in vivo (inside the body).
      ::基因疗法、将基因插入人体细胞以治愈基因紊乱,可以是体外(身体外)或体外(身体内)。
    • Gene therapy is still in the experimental stages, but some trials have been successful.
      ::基因疗法仍处于试验阶段,但有些试验是成功的。

    Explore More
    ::探索更多

    1. What defect causes symptoms in patients with cystic fibrosis?
      ::哪些缺陷导致患细胞纤维化症的患者出现症状?
    2. What are some challenges for using viruses to transfer DNA to cells?
      ::使用病毒将脱氧核糖核酸转移到细胞方面有哪些挑战?
    3. Besides viruses, what are some other methods to transfer DNA to cells?
      ::除了病毒,还有什么其他方法可以把DNA转移到细胞?

    Review
    ::回顾

    1. What is gene therapy?
      ::什么是基因疗法?
    2. Could gene therapy someday cure the common cold? Why or why not?
      ::基因疗法有一天能治愈普通的寒冷吗?
    3. What's the difference between ex vivo and in vivo gene therapy?
      ::异活基因疗法和异活基因疗法有什么区别?
    Editor526&18