Name: 基於 CRISPR 的基因編輯技術掀起了一場醫學革命--下一步會是什麼？ (CRISPR-based gene editing revolutionized medicine—what's next?)
Uploaded: 2025-06-18T17:59:32.000Z
Duration: 5 min 10 s

Recent technological advancements have paved the way for the development of medical treatments for life-threatening diseases.

最近的技術進步為開發治療危及生命的疾病的藥物鋪平了道路。

At the forefront of this is CRISPR-Cas9, a gene editing technology elucidated over a decade ago and honored with the Nobel Prize in Chemistry in 2020.

CRISPR-Cas9就是其中的佼佼者，這項基因編輯技術早在十多年前就已被闡明，並於2020年榮獲諾貝爾化學獎。

I traveled to Berlin to meet Emmanuel Charpentier, one of CRISPR-Cas9's inventors.

我前往柏林會見了CRISPR-Cas9的發明者之一埃馬紐埃爾-沙爾彭蒂耶（Emmanuel Charpentier）。

CRISPR is a system that exists in bacteria and allows bacteria to defend themselves against infection by viruses.

CRISPR 是一種存在於細菌中的系統，能讓細菌抵禦病毒感染。

So bacteria can also be infected by viruses and they have evolved different immune systems and one of them is the CRISPR-Cas9 system.

是以，細菌也會被病毒感染，它們進化出了不同的免疫系統，其中之一就是 CRISPR-Cas9 系統。

And so by our interest in the lab to find new molecules that could have a role in the bacterium Streptococcus pargenes, we came across a very neat mechanism that allows us to really recognize the virus that infects the bacterium in a very, very specific manner at the level of the genome of the virus.

是以，我們在實驗室中發現了一種新的分子，這種分子可以在副鏈球菌中發揮作用。我們發現了一種非常巧妙的機制，它可以讓我們在病毒的基因組水準上，以一種非常非常特殊的方式真正識別出感染細菌的病毒。

And we exploited this natural mechanism to develop the CRISPR-Cas9 technology.

我們利用這一自然機制，開發出了 CRISPR-Cas9 技術。

With a mission to cure disease, in 2013 Emmanuel co-founded CRISPR Therapeutics, a biotech company that uses CRISPR technology to develop gene therapies.

帶著治癒疾病的使命，埃馬紐埃爾於 2013 年共同創立了 CRISPR Therapeutics 公司，這是一家利用 CRISPR 技術開發基因療法的生物技術公司。

After 10 years in operation, CRISPR Therapeutics and its Vertex Pharmaceuticals saw their drug Casgivy become the first CRISPR gene therapy to be approved by the FDA.

經過 10 年的運作，CRISPR Therapeutics 及其 Vertex Pharmaceuticals 公司的藥物 Casgivy 成為首個獲得美國食品及藥物管理局準許的 CRISPR 基因療法。

It treats two serious inherited blood disorders, transfusion-dependent beta thalassemia and sickle cell disease.

它可以治療兩種嚴重的遺傳性血液疾病，即輸血依賴型β地中海貧血症和鐮狀細胞病。

We spoke with the CEO of CRISPR Therapeutics, Samath Kulkarni, at the company's headquarters in Boston.

我們在波士頓的公司總部採訪了 CRISPR Therapeutics 的首席執行官 Samath Kulkarni。

In the U.S. there are about 100,000 patients that suffer from sickle cell disease.

美國約有 10 萬名鐮狀細胞病患者。

Nearly a quarter of them are severe sickle cell patients that have pain every day, that have to be hospitalized every so often, and ultimately don't live as long as everyone else.

其中近四分之一是嚴重的鐮狀細胞患者，他們每天都很痛苦，不得不經常住院治療，最終也無法像其他人一樣長壽。

With Casgivy we're taking the bone marrow cells from the patient, making the edit for that particular patient, and we're putting it back into the patient.

有了卡斯吉維，我們從病人身上提取骨髓細胞，針對特定病人進行編輯，然後再把它放回病人體內。

And it reconstitutes the hematopoietic system of the patient.

我們正在為你專門研製一種藥物。

CRISPR Therapeutics currently has seven clinical and 10 preclinical programs in the fields of oncology, autoimmune cardiovascular disease and diabetes.

CRISPR Therapeutics 目前在腫瘤、自身免疫性心血管疾病和糖尿病領域有 7 個臨床項目和 10 個臨床前項目。

By 2027 it hopes to have enough data to launch the next wave of therapies, most of which will be created in these labs.

到 2027 年，它希望能獲得足夠的數據來推出下一波療法，其中大部分將在這些實驗室中誕生。

Shaheen Kabir is CRISPR Therapeutics principal scientist.

Shaheen Kabir 是 CRISPR Therapeutics 首席科學家。

The first part of developing a therapy is figuring out what gene or target sequence is causative of disease and what you want to basically target to correct or repair.

開發療法的第一部分是找出致病基因或靶序列，以及你想要糾正或修復的基本靶點。

The second part is then designing components that will allow you to go to that gene sequence and perform the edit of interest.

第二部分是設計組件，使您能夠進入該基因序列並執行感興趣的編輯。

Once you've developed these components you now have to figure out how to deliver them to the cells or the tissues that you're interested in.

開發出這些成分後，您現在必須想辦法將它們輸送到您感興趣的細胞或組織中。

And then finally these pieces all come together and you would assess the efficacy and the potency of the therapy that you've developed.

最後，這些部分全部整合在一起，你將評估所開發療法的療效和效力。

CRISPR gene therapies can be administered in two ways.

CRISPR 基因療法可以通過兩種方式進行。

Ex vivo, the most common practice, is when cells are removed from the patient's body, edited and then reintroduced.

體外療法是最常見的做法，即從病人體內取出細胞，經過編輯後重新輸入。

The alternative method is in vivo, when changes to a cell's genetic material are made inside the patient's body, something CRISPR Therapeutics is working to make more widely available.

另一種方法是體內療法，即在患者體內改變細胞的遺傳物質，CRISPR Therapeutics 正在努力使這種方法得到更廣泛的應用。

The scientists here are exploring how CRISPR components can reach hard-to-target tissue such as those in the brain or lungs for in vivo editing.

這裡的科學家們正在探索 CRISPR 組件如何能夠到達難以瞄準的組織，如大腦或肺部組織，進行體內編輯。

To investigate and develop new therapies, the company set up another research team called CRISPR-X.

為了研究和開發新的療法，公司成立了另一個名為 CRISPR-X 的研究團隊。

There are a lot of diseases that we're not currently able to target with CRISPR and that may be because of delivery, we're not able to get the right components to the right place, but it might also be that the kind of change that we need to make, we don't necessarily have the tools for yet.

有很多疾病我們目前還無法用CRISPR技術進行靶向治療，這可能是因為傳遞的原因，我們無法把正確的成分送到正確的地方，但也可能是因為我們需要做出的改變，我們還不一定有工具來實現。

And that's what the focus of CRISPR-X is.

CRISPR was developed to advance medicine but its groundbreaking capabilities have led to its use beyond healthcare, including biotechnology and agriculture.

開發 CRISPR 的目的是為了推動醫學發展，但其突破性的功能已使其應用超出了醫療保健領域，包括生物技術和農業。

I think it's always important to figure out, you know, what are the needs for the population and then whether CRISPR is the right technology to use and if this comes together then it's important to, you know, to consider new technologies.

我認為，重要的是要搞清楚，你知道，人們的需求是什麼，然後再看CRISPR是否是正確的技術，如果是，那麼考慮新技術就很重要了。

So looking into your crystal ball, Emmanuelle, where do you see the future of this technology including with CRISPR-X?

埃瑪紐埃爾，從你的水晶球中，你認為這項技術（包括CRISPR-X）的未來在哪裡？

So we will see the use of the technology in the future, directly or indirectly, being important for other diseases that will be cured.

是以，未來我們將看到，這項技術的使用將直接或間接地對其他疾病的治癒起到重要作用。

So either using CRISPR or new therapeutics that would have come up on the market thanks to CRISPR because CRISPR was used in the process of research and development.

是以，無論是使用CRISPR，還是在研發過程中使用CRISPR，市場上都會出現新的療法。