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    Home > Active Ingredient News > Study of Nervous System > The first child with dual route gene therapy!

    The first child with dual route gene therapy!

    • Last Update: 2021-05-22
    • Source: Internet
    • Author: User
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    Recent popular reports from Yimaike ★ Nanjing · Big coffee gathered in May2021 Nucleic Acid Drug Development Forum Announcement Schedule ★ May Nanjing2021 Nucleic Acid Drug Development Forum is about to open May 6, 2021 / Yimai Ke News eMedClub News/--A U.
    S.
    Authority The newspaper USA TODAY reported a story about gene therapy and a child with Sandhoff's disease-Now, instead of certain death, she faces an uncertain future.

    Alissa is a 14-month-old Danish girl.
    For the first four months, Alissa, like all small lives, worked hard to roll, sit up, and play with her parents with toys.

    But the bad news came inadvertently.
    Between the doctor's assurance of health and the diagnosis of Sandhoff, Alissa's parents felt like a roller coaster.

    In the eighth month of the collapse, Alissa was admitted to the hospital and was diagnosed with Sandhoff.

    Sandhoff disease, also known as GM2 ganglioside deposition disease, is a rare lysosomal storage disease, which is an autosomal recessive inherited disease.
    Hexosaminidase is caused by mutations in the β-subunit gene of hexosaminidase (HEXB).
    Both A and hexosaminidase B are deficient, and GM2 ganglioside is deposited in the central nervous system and the lysosomes of somatic cells, causing a series of clinical symptoms.

    Fortunately, gene therapy is no longer a rare and novel thing in Europe and America.
    It has created too many miracles and is becoming more and more common.

    Alissa's father searched the Internet for "gene therapy + Sandhoff", and he found that there was an experimental Sandhoff gene therapy at the University of Massachusetts.

    In late January 2021, 14-month-old Alissa received gene therapy.

    This treatment method will deliver healthy and functional genes to Alissa’s brain target cells, allowing the cells to recognize the genes to encode the missing enzymes, eliminate disease-causing proteins, and ultimately aim to alleviate/reduce disease progression .

    Different from other children in the test, after Alissa was tested, the researchers believed that Alissa was eligible for the enrollment of patients.
    Alissa was young and the disease had not caused greater and irreversible damage to Alissa.
    Gene therapy is expected to help Alissa.
    Eliminate these disease-causing lysosomal storage, while giving a new way of administration and dosage levels.

    The researchers said that the best way to administer the drug is through the thalamus and re-administered with spinal cord injection.
    Such administration and dosage levels will be able to guide her entire nervous system and brain cells to start producing the required enzymes.

    The first two children who received this gene therapy had lower doses.
    One was given through the thalamus and the other was given through spinal cord injection.
    Alissa was a gene therapy patient who received both routes of administration at the same time.

    Dr.
    Terence Flotte, Dean of the University of Massachusetts School of Medicine, said: “Alissa’s immune system is controlled by drugs.
    If needed in the future, she can receive viral gene therapy again.

    ” said Dr.
    Pavan Cheruvu, CEO of Sio Gene Therapies: “In fact We don’t need so much enzyme activity to make neurons function.

    "One week after the operation, Alissa’s eyes were able to roll and focus steadily, although she still couldn’t see anything; two weeks after the operation, Alissa started smiling again.
    , And laughter from the abdomen; in February of this year, Alissa could consciously move her hands and eat more; recently, Alissa learned to suck bottles.

    Currently, Alissa is undergoing rehabilitation therapy to re-perceive and control her body, and it seems that everything is moving towards a positive future.

    Doctors are also admonishing not to expect too much.
    No one knows what kind of future gene therapy will bring. At the end of the article, Alissa's parents said that she may never be like a normal child, but if she doesn't try hard, Alissa won't even have the opportunity to communicate with us in a wheelchair/other things.

    Sandhoff is a rare disease.
    According to the statistics of the World Health Organization, there are about 5000-8000 rare diseases worldwide.

    Adding these patients together, the total number exceeds 300 million.

    In other words, 5 out of every 100 people are affected by rare diseases, and these patients urgently need new treatment options.

    There was no cure in the past.
    Since gene therapy has been successful in a variety of rare/genetic diseases, the world seems to show them a glimmer of hope for this group of patients.

    But at present, gene therapy is still in an emerging stage of rapid development, requiring more research and more verification.

    But there is no doubt that the future will be better! The 2021 nucleic acid drug development forum will kick off in Nanjing from May 7th to 8th, 2021, to welcome the "new era of super drugs".
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