Questions tagged [gene-therapy]

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What are the potential dangers (if any) facing the twin girls recently born in China with their CCR5 gene modified?

According to this Nature news article, a Chinese researcher claims to have made the world's first genome-edited baby using the popular CRISPR–Cas9 genome-editing tool. A gene called CCR5 was modified ...
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23 views

How does a CRISPR therapy work?

Afaik you can use CRISPR to edit genes of a cell. But how should therapy for a human work based on CRISPR? Can a drug perform it on vast numbers of ill cells?
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2answers
260 views

What is the difference between “gene delivery” and “gene therapy”?

What is the specific difference between gene delivery and gene therapy? As far as I understand, gene delivery is the first step of gene therapy, but where does gene delivery end and gene therapy ...
-1
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1answer
81 views

which of these fields will cure aging? [closed]

life extension is expected to happen as a result of breakthroughs in tissue rejuvenation, stem cells, regenerative medicine, molecular repair, gene therapy, pharmaceuticals, and organ replacement. ...
2
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0answers
18 views

Condition for a genetically modified group of cell to remain and to spread?

I was wondering: when a treatment modifies a group of gene (like in gene therapy), what makes a group of cell remains? I mean, when a cell divide itself to create new ones, if a cell was initially ...
2
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1answer
33 views

Using viruses to treat altered or misconfigured DNA

Consider how a Retrovirus can modify existing cell DNA to 'execute instructions' on its behalf. I wondered: Why can we not utilize lab-generated viruses to infect sick patients with a 'healthy' ...
6
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2answers
211 views

How would you effect bulk DNA gene therapy for a human?

Let's imagine that we understood DNA programming and our genome very well and realized that there were some significant flaws (we die, we need sleep, etc.) And let's imagine that we understand how to ...
0
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1answer
42 views

Possible complications due to gene-therapy?

Edward Lanphier, CEO of Sangamo BioSciences mentions here(https://youtu.be/dJ1B8XnyEnM?t=1215) that scientists can now specifically target cells, change the genome within a cell. Say you want to cure ...
1
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2answers
597 views

Risks in bacterial phage therapy

I just finished reading J. Craig Venter's book Life at the Speed of Light: From the Double Helix to the Dawn of Digital Life. The book is a little over a year old now, and Venter has an optimistic ...
3
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0answers
110 views

In vivo delivery of ssDNA

I am looking for a system with which I can deliver (large quantities of) ssDNA in vivo (say in mice). I do not know whether it is of importance but the 5' end of the ssDNA fragments are assumed to be ...
0
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1answer
152 views

Current state of Gene Therapy [closed]

I am interested in learning about attempts to treat adult individuals suffering from a genetic disease in which the underlying changes in the DNA of the gene are understood. (i) Are there approaches ...