In layman's terms: She had a particularly vicious form of cancer where the very cells that are supposed to protect your health -- your T-cells -- are the enemy. So they made three edits to donor cells and wiped out her compromised cells. Then gave her a bone marrow transplant afterwards to replenish her immune system with new cells.<p>I'm squeamish about genetic research, but this looks really good to me. I hope she recovers fully and there are no further complications, though I imagine it's too early to guess what her prognosis might be.<p><i>So there are already trials of base editing under way in sickle-cell disease, as well as high cholesterol that runs in families and the blood disorder beta-thalassemia.</i><p>That's a big deal for people with those genetic disorders.
> The team at Great Ormond Street used a technology called base editing, which was invented only six years ago.<p>Fun fact: J M Barrie who wrote Peter Pan donated the copyright of both the book and play in perpetuity to Ormond Street children's hospital. It's been a significant source of funding for them over the years.
In response to the multiple people asking if base editing and CRISPR are the same, the answer is: not quite. Same idea, different enzyme.<p>The enzyme used by CRISPR is Cas9 (which is why you'll often see it referred to as CRISPR-Cas9), which acts like a pair of molecular scissors and cuts strands of DNA at a specific point in the genome.<p>Base editing is a newer technique and utilizes an enzyme called deaminase, which can change the chemical structure of a base pair without doing the "cutting" part. There are specific deaminases based you use depending on which base pair you're trying to modify.<p>To oversimplify a bit: base editing is a more precise version of CRISPR, which is less likely to cause more changes than actually desired, as is a problem with CRISPR. That doesn't make CRISPR obsolete though, because if your goals actually <i>is</i> to remove entire portions of a genome, CRISPR is a more effective technique. Regardless, there is a lot of overlap between the methods: both usually use adenoviruses as the delivery vehicle, you can use both enzymes in tandem, etc.
Great to see medical techniques slowly starting to resemble engineering design:<p>1. Donor T-cells added with receptors to destroy the recipient's cancerous T-cells.<p>2. Donor T-cells' markers removed to prevent them from attacking each other.<p>3. Donor T-cells' existing receptors removed to prevent them from attacking the recipient's other cells (since the donor T-cell will see them as 'foreign').<p>4. Donor T-cells altered to resist chemotherapy.<p>It seems each of these changes were achieved by editing specific base pairs to <i>break</i> the relevant gene, except perhaps for (1)
In heroic medical science history fiction it's a bit of a trope to write "the operation was a success but the patient died"<p>I sincerely hope she survives at least 5 years, and significantly more. If she gets only a single year of treatment -free high quality life outside of respite care, that would be a bitter pill outcome but might still be held a success. Sometimes giving people a year of quality life at end-of-life is a good outcome.<p>The article as written is light on for data and time info. She's had at least 3 post treatment checks one of which was 3 months, the other 2 not stated but 6monthly might not be unusual.<p>Recurrences in blood borne diseases are not unheard of. She had some signs of recurrence at 3 months but it went away. Maybe fragment traces? Non viable remainders flushing out of the marrow? (I don't know if this is even plausible. I'm not in medicine)<p>It's great news, but so was Christian Barnard's first transplant. In modern terms the survival wasn't very long. (200 days in the first 4)<p>A better outcome would be like Steptoe's work on assisted conception and birth. (IVF) and I certainly hope she and future patients have that.
How did they remove / destroy the T-cell killing T-cells before the the 2nd marrow transplant?<p>Otherwise, it would seem they (the engineered T-cells) would hang around, and destroy the T-cells created by the marrow transplant.
It sounds like this is a continuation of work that was tested as far back as 2015: <a href="https://www.businessinsider.com/r-gene-edited-cells-keep-cancer-babies-well-more-than-one-year-on-2017-1?amp" rel="nofollow">https://www.businessinsider.com/r-gene-edited-cells-keep-can...</a>
Very similar report by the same author from 2015: <a href="https://www.bbc.com/news/health-34731498" rel="nofollow">https://www.bbc.com/news/health-34731498</a><p>It doesn’t explain what “designer immune cells” were, so it’s hard to tell what’s different in this more recent treatment.
This looks pretty great, hopefully a huge step forward for the medical industry and finding cures for cancer. Unfortunately don't know too much about it; wish there was something that allowed me to learn about medical problems easily, without needing to understand the jargon
We are at the "assembly" stage of genetic tinkering. Manually changing over the 'machine code' of the cell, the linear sequence of physically bound instructions. I wonder what the future might bring in terms of higher level programming of cells.
For clarity’s sake,<p>> “Base editing allows scientists to zoom to a precise part of the genetic code and then alter the molecular structure of just one base, converting it into another and changing the genetic instructions.”<p>can be reduced to “gene editing.”
When is the head of the department going to do a televised interview?<p>> <a href="https://youtu.be/B3xY6Ffy_wE?t=68" rel="nofollow">https://youtu.be/B3xY6Ffy_wE?t=68</a>
I’ve started watching star-trek (as it makes me fall asleep immediately) - this stuff sounds like a star-trek episode!<p>Something Dr Crusher would come up with.<p>It makes a distant future where we quickly engineer a fix and inject it seem almost plausible.
This is a wonderful outcome and amazing science and all involved are to be commended for saving this girl's life.<p>However would it not make more sense to try and prevent the disease in society, since there is a viral component to it? It is a retro-virus and was first discovered in 1977. Is there even research on a vaccine? We have one for cervical cancer. (Hit's a nerve since I have a child with Lymphoma)<p><a href="https://www.who.int/news-room/fact-sheets/detail/human-t-lymphotropic-virus-type-1" rel="nofollow">https://www.who.int/news-room/fact-sheets/detail/human-t-lym...</a>
Tangential, but I wonder how long it'll be before we start combining these recent advances in AI with a dataset like Medikanren and identifying really custom treatments.
I'm not sure it counts as "revolutionary" if it has helped only 1 person, and maybe as many as 10-12 per year. It's really the opposite of revolutionary, though I am very happy for this family and their daughter.