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CRISPR

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  1. Stefaan_ 16 april 2018 11:43
    rispr sees gene therapy milestone in Europe
    Apr. 16, 2018 3:10 AM ET|By: Yoel Minkoff, SA News Editor

    Humans are set to be genetically modified for the first time in Europe after regulators gave the go ahead to trial Crispr Therapeutics' (NASDAQ:CRSP) DNA-splicing therapy.

    A destructive blood disorder known as beta thalassaemia could be cured by altering the body's code and restoring healthy levels of haemoglobin.

    "We will look back and think that this is the real beginning of gene therapy," said Prof. Robin Lovell-Badge of London's Francis Crick Institute.

    Now read: Time Out: Talking Strategy For CRISPR's Big 3 »
  2. MarkvB 7 januari 2021 20:24
    Imara declined yesterday as its sickle cell treatment appears to be not at par with its peers

    Jan. 07, 2021 7:36 AM ETIMARA Inc. (IMRA)By: Vandana Singh, SA News Editor
    Imara (NASDAQ:IMRA) closed in red, down 38% yesterday as investors apparently saw the company's sickle cell data lacked benefit when compared to its peers.
    Data showed that IMR-687 had no meaningful impact on key measures like fetal hemoglobin ((HbF)) or hemoglobin levels when taken by itself. Combination with HU showed slight benefits in F- cells and fetal hemoglobin levels but hemoglobin decreased on average after 24 weeks.
    Three of eight combination patients had absolute increases in HbF percentage of greater than 1%, with a mean absolute increase in HbF percentage of 4.3% in that subset of patients
    Global Blood Therapeutics and Novartis already have approved therapies to treat sickle cell patients while one-time treatments from Bluebird bio (NASDAQ:BLUE) and Vertex/Crispr (NASDAQ:VRTX) (NASDAQ:CRSP) have shown promise for potentially curative therapies, raising the bar.
    SVB Leerink analyst Joe Schwartz defended the company stating that Covid-19 hurt the trial because of missed visits that “potentially undermined the quality of the data”.
    He also cited that the drug doses examined are much lower than those expected in future trials, and he believes that “longer and higher dosing” could lead to improved efficacy.
    SVB Leerink said in a note "Imara’s Phase 2a data announced Wednesday represents a “rearward looking view of IMR-687’s development” and isn’t likely a good indicator of what’s to come in future readouts". Sees the stock’s weakness as a potential buying opportunity ahead of IMRA’s multiple data readouts in 2021.
    Imara shares are up 3% in premarket trading today.
  3. MarkvB 7 januari 2021 22:46
    CRISPR doubles lifespan of mice with rapid ageing disease progeria







    HEALTH 6 January 2021
    By Michael Le Page
    A cell from a person with progeria
    A cell from a person with progeria
    ALVIN TELSER/SCIENCE PHOTO LIBRARY

    CRISPR gene editing has been used to more than double the lifespan of mice engineered to have the premature ageing disease progeria, also greatly improving their health.
    The results far surpassed expectations. Progeria affects many different organs in the body, and the team behind the work didn’t expect that correcting the mutation in a relatively low proportion of cells – 10 to 60 per cent – would have such a big effect. “We were quite amazed,” says David Liu at Harvard University.
    Hutchinson-Gilford progeria syndrome is a rare condition caused when a mutation, which probably took place in the testes or ovaries of a child’s parents, results in a single DNA letter change in one of the two copies of the gene for the lamin A protein. This leads to the production of an abnormal protein called progerin that interferes with cell division and causes many symptoms of premature ageing. The average lifespan of children with progeria is 14 years.
    Advertisement
    Conventional gene therapy, which involves adding genes, cannot help. People with progeria still have one healthy copy of the lamin A gene – the problem is the mutant progerin protein.
    The standard form of CRISPR gene editing, which involves cutting DNA with the Cas9 protein, can be used to disable the mutant gene. The trouble is that it often disables the healthy copy too, as well as causing other unwanted changes.
    Liu’s team has been modifying the Cas9 protein so instead of cutting DNA, it changes one DNA letter to another, a process known as base editing. He and his colleagues have now used a CRISPR base editor to correct the single-letter change that causes almost all cases of progeria, first in skin cells taken from a person with progeria and then in mice with a human version of the lamin A gene.
    Read more: The powerhouses inside cells have been gene-edited for the first time
    A virus carrying the genes for the base editor was injected into the blood of 2-week-old mice – roughly equivalent to 5-year-old children, says Liu – with the progeria mutation. A single injection boosted the median lifespan from 215 to 510 days, and the treated mice were also far more active.
    Because the mice had the human gene, exactly the same approach could be used in human trials. However, Liu’s team has already developed even more efficient base editors.
    In November 2020, the US Food and Drug Administration approved the first-ever drug for treating progeria. In trials, it increased lifespan by an average of 2.5 years over the maximum follow-up time of 11 years. Liu thinks combining this drug with the CRISPR base editing will work well.
    The findings also boost hopes that many other conditions could be treated through base editing. Half of all known disease-causing mutations involve a single-letter change, most of which can be corrected with existing base editors, says Liu.


    Read more: www.newscientist.com/article/2264168-...
  4. Scoupy- 16 januari 2021 11:07
    Goedemorgen,

    Sinds kort ben ik geïnteresseerd geraakt in Crispr en wellicht nog andere bedrijven die DNA kunnen aanpassen.
    Ik zie in deze (bio hacking) technologie wel toekomst, verder zal ik mij nog wat meer moeten inlezen in deze wereld.

    - Hoever zijn ze in het proces?
    - Zijn er licenties?
    - Wordt het gereguleerd?
    - Kosten?
    - En al het andere wat van belang is

    Ik kan mij voorstellen dat overheden niet happig zijn op deze technologie.
    Het kan volgens mij ziektes en of aandoeningen aanpakken via DNA hacking maar dit kan natuurlijk ook toegepast worden voor foute doeleinde, het perfectioneren van de mens, blauwe ogen, meer spiergroei en noem maar op.

    Of heb ik het fout?

    Groeten!
  5. Biopharma 18 juni 2021 08:41
    quote:

    MarkvB schreef op 7 januari 2021 22:46:


    CRISPR doubles lifespan of mice with rapid ageing disease progeria







    HEALTH 6 January 2021
    By Michael Le Page
    A cell from a person with progeria
    A cell from a person with progeria
    ALVIN TELSER/SCIENCE PHOTO LIBRARY

    CRISPR gene editing has been used to more than double the lifespan of mice engineered to have the premature ageing disease progeria, also greatly improving their health.
    The results far surpassed expectations. Progeria affects many different organs in the body, and the team behind the work didn’t expect that correcting the mutation in a relatively low proportion of cells – 10 to 60 per cent – would have such a big effect. “We were quite amazed,” says David Liu at Harvard University.
    Hutchinson-Gilford progeria syndrome is a rare condition caused when a mutation, which probably took place in the testes or ovaries of a child’s parents, results in a single DNA letter change in one of the two copies of the gene for the lamin A protein. This leads to the production of an abnormal protein called progerin that interferes with cell division and causes many symptoms of premature ageing. The average lifespan of children with progeria is 14 years.
    Advertisement
    Conventional gene therapy, which involves adding genes, cannot help. People with progeria still have one healthy copy of the lamin A gene – the problem is the mutant progerin protein.
    The standard form of CRISPR gene editing, which involves cutting DNA with the Cas9 protein, can be used to disable the mutant gene. The trouble is that it often disables the healthy copy too, as well as causing other unwanted changes.
    Liu’s team has been modifying the Cas9 protein so instead of cutting DNA, it changes one DNA letter to another, a process known as base editing. He and his colleagues have now used a CRISPR base editor to correct the single-letter change that causes almost all cases of progeria, first in skin cells taken from a person with progeria and then in mice with a human version of the lamin A gene.
    Read more: The powerhouses inside cells have been gene-edited for the first time
    A virus carrying the genes for the base editor was injected into the blood of 2-week-old mice – roughly equivalent to 5-year-old children, says Liu – with the progeria mutation. A single injection boosted the median lifespan from 215 to 510 days, and the treated mice were also far more active.
    Because the mice had the human gene, exactly the same approach could be used in human trials. However, Liu’s team has already developed even more efficient base editors.
    In November 2020, the US Food and Drug Administration approved the first-ever drug for treating progeria. In trials, it increased lifespan by an average of 2.5 y tweakbox.mobi/ ears over the maximum follow-up time of 11 years. Liu thinks combining this drug with the CRISPR base editing will work well.
    The findings also boost hopes that many other conditions could be treated through base editing. Half of all known disease-causing mutations involve a single-letter change, most of which can be corrected with existing base editors, says Liu.


    Read more: www.newscientist.com/article/2264168-...

    issue got solved!!
  6. forum rang 5 wiegveld 28 juni 2021 23:00
    Bloomberg:
    See below

    Gene editing stocks are starting the week with a big advance following a major breakthrough for the industry. Early trial data from Intellia Therapeutics (NASDAQ:NTLA), whose shares are up 33% premarket to $117, released interim data from a phase 1 trial of a CRISPR candidate showing the ability to genetically edit cells inside a liver. The data was presented today at the 2021 Peripheral Nerve Society Annual Meeting and in the New England Journal of Medicine.

    What happened? CRISPR technology, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, was previously restricted to editing cells outside the body or in the eye. It also faced challenges like sticking molecular scissors into the body or slicing DNA in a select number of tissues. This time around, researchers injected a CRISPR drug into the blood of people born with transthyretin amyloidosis, a destructive disease that causes fatal nerve and heart disease. The results showed that the editing technology was able to nearly shut off production of the toxic protein generated by their livers by knocking the gene's activity.

    While it's too early to tell whether the CRISPR treatment will ease symptoms of the disease, or if other problems will surface over time, there's still a lot to be excited about. "The allure and the promise of CRISPR is this notion that you can change any gene, anyhow, anywhere in the genome, so long as you can get it there. And that last proviso is the key one," declared Intellia CEO John Leonard. "This is the first time CRISPR has ever been infused into a patient and the first time we've been able to target a gene successfully."

    On the move premarket: Beam Therapeutics (NASDAQ:BEAM) +12%; Editas Medicine (NASDAQ:EDIT) +11%; CRISPR Therapeutics (NASDAQ:CRSP) +8%; ARK Genomic Revolution ETF (BATS:ARKG) +2.5%; Regeneron (NASDAQ:REGN) +1.7%.
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