Questions tagged [gene-therapy]

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Databases medicine and therapy - animal & human studies, technology readiness level

I am looking for some universal databases that would offer information about drugs and therapies currently in development. The area of interest is mostly cell and gene therapy. I am looking for ...
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Can In Vivo Gene Therapy Cause allorecognition/Rejection?

Organ rejection from organ transplants is least common when donors and receivers are genetically related; As I understand it, this is because the antigens on the surface of the cells of the donated ...
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Can an injection of unipotent stem cells cause teratoma?

I understand that injecting pluripotent stem cells causes formation of teratomas. Does injecting unipotent stem cells also cause teratoma? If it does not form teratoma, is the injection of unipotent ...
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Why doesn't HIV give you cancer if retroviral gene therapy gives you cancer?

More than twenty patients have been treated in France and Britain, with a high rate of immune system reconstitution observed. Similar trials were restricted or halted in the US when leukemia was ...
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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 ...
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Do bacteria duplicate the copied virus DNA to put into a Cas9 protein when fighting the virus off again?

When bacteria insert a part of invading virus DNA into its own genetic sequence, on the 2nd invasion does it duplicate that copied DNA again from wherever the bacteria placed it and put it into a Cas9 ...
3 votes
2 answers
270 views

Can the spike protein created by the Covid mRNA vaccines be created independently of the human body, and is there a higher cost to that?

How different in principle is using the bodies own mRNA to create the coronavirus spike protein differ from other methods of using genes to manufacture other drugs or proteins and is there a cost ...
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1 answer
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Convert fasta to ascii?

Is there an algorithm or tool to convert a fasta sequence file (such as the file linked below) into an ascii file for the purpose of gene synthesis? https://www.rcsb.org/fasta/entry/6EQJ To the best ...
12 votes
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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 ...
2 votes
1 answer
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What advantages could there be for using the unseen variant chosen by He Jiankui instead of the naturally occurring CCR5-∆ 32 mutation?

Jennifer Doudna mentioned in https://youtu.be/9Yblg9wDHZA?t=2566 on 2019-02-21 that He Jiankui introduced an unseen variant of the CCR5 gene when gene editing the twin humans Lulu and Nana. What ...
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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. ...
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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 votes
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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' ...
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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 ...
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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 vote
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685 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 votes
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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 ...
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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 ...