First human body gene editing treatment implementation in the United States, is expected to cure the hunter’s syndrome

44 years old American man Brian & middot; Madeleine (Brian Madeux) bearing the weight of the node of change in human history. According to the Associated Press, November 13, Madeleine with hunter’s syndrome at the university of California, San Francisco, Mr Benioff at children’s hospital accepted an audacious treatment: genes in the body. This is the first time in the world by editing, in vivo gene therapy of genetic diseases.

a suit of casual clothes mudd pudgy, treatment on the same day, he wore a cap to keep out high convex head due to illness. Seemingly mudd just lying in bed, had a three hours of the infusion, but from the iv into mudd vein transparent liquid is recording the human no medicine can cure genetic disease against the determination. Transparent liquid contained adeno-associated virus (AAV), zinc finger nuclease (ZFN) and Madeleine was born lack normal IDS genes, they will work for some kind of unprecedented.

Brian & middot; Madeleine (Brian Madeux) is undergoing treatment. Source: the Associated Press.

and CAR – T remove cells in patients with modified feedback, or functional protein expression in vivo using AAV import cDNA gene therapy, Madeleine treatment more thoroughly, this time his genome information will be a permanent change.

a month later, the treatment is effective to have a preliminary answer. Three months later, the safety and efficacy of the therapy will be further confirmed.

it is worth mentioning that Madeleine gene editing tool is used by the treatment of zinc finger nuclease, rather than the fire & other Gene demons cut & throughout; The CRISPR. Compared with & other; Epigenetic & throughout; CRISPR, scientists study of zinc finger nuclease can be traced back to the 1980 s, is considered to be the first generation of gene editing tools. Although the operation of the zinc finger nuclease is more complex, but due to locate the sequence of longer, gene editing precision is also higher. The U.S. food and drug administration (FDA) has approved a zinc finger nuclease used in human clinical trials, the CRISPR did not be allowed.

Madeleine is holy and companies in the United States (Sangamo Therapeutics) code & other; SB – 913 & throughout; In the research of treatment of the first human clinical trials. In advance before the test to get the United States national institutes of health (NIH) related advisory committee for permission.

st and listed company won a series of key patents have zinc finger nuclease, it plans to expand the clinical trial samples, tested in more adult patients, ultimately achieve the goal of treatment in children patients. Although genes editor can help mudd solution from the root causes, but his damage has been unable to make up for the past 40 years, so from childhood to intervene treatment more effective.

hunter’s syndrome

hunter’s syndrome is a rare genetic disease. Mainly male disease, every 100000 to 170000 in newborn boys will have an unfortunate suffering from hunter’s syndrome.

every hunter’s syndrome patients lack of IDS, and not have the presence of the gene, its job is to generate an enzyme that can decompose toxic carbohydrates. Because of the lacking of IDS genes, this play & other; The cleaner & throughout; The role of the absence of enzymes, cause cells to accumulation of toxic metabolite. These and other Garbage & throughout; More and more, their devastating to the patient’s organs.

most of the patients died before the age of 20, by contrast, Madeleine was relatively moderate. In spite of this, Madeleine has experienced more than 20 times large and small operation. Last year a bronchial inflammation and pulmonary inflammatory nearly took his life, his windpipe has been distorted, secretions to dredge through coughing.

more unfortunately, due to the hunter’s syndrome patients is on the genome appears incomplete, doctors were baffled. Weekly to the patient’s body is the only alleviate means of input the missing enzyme. As a tactic, the cost of enzyme replacement therapy in the $100000 to $400000 per person per year.

& other; Lack what fill what & throughout;

& other; We cut off their DNA, open a vent, insert a (normal) gene, and sewing. Can’t you see traces of repair, it will be part of your DNA, spend the rest of my life with you. Throughout the &; St and CEO sandy & middot; McRae (Sandy Macrae) in an interview with the Associated Press, so to describe their therapy.

since the hunter’s syndrome is missing IDS gene, is the radical way & other; Lack what fill what & throughout; .

from vein into the mudd, not pathogenic adeno-associated virus (AAV) act as & other; Cars & throughout; , will carry the two zinc finger nuclease and one normal gene, direct mudd liver cells. & other; Cars & throughout; Is designed only to liver this & other; Destination & throughout; . After arrived in liver cells, zinc finger nuclease first open GPS, accurately find & other; Operating point & throughout; And then incarnation & other; Scissors & throughout; Cut, double-stranded DNA, filling into the normal gene. Through DNA repair mechanisms, the original DNA fragments to & other; Accommodate & throughout; The new normal gene.

according to introducing, need only 1% of the liver cells receive new normal gene, Matthew hunter’s syndrome can get effective treatment.

for Madeleine, even if the effective treatment, the disease in the past few decades of the damage is irreversible. In addition, the journal science reports pointed out that the gene therapy and the plight of enzyme replacement therapy as possible: not through the blood brain barrier, prevent many substances from the blood into the brain tissue of barrier. This means that Madeleine brain damage may not be able to get stem.

ups and downs of the gene therapy

although the safety of the zinc finger nuclease after repeated tests, the therapy before tested mudd, also has good validation in animal models. But when it comes to gene therapy, people still can’t embrace with light heart, it is quite safe?

this concern is not without reason. At one time in the early 1990 s, gene therapy has become the darling of the scientific community, many human trials hot. But at the cost of life & other; Accident & throughout; Pours cold water for gene therapy.

in 1999, with bird amine acid carbamyl acyltransferase deficiency of the 18-year-old boy Jessie & middot; Kissinger (Jesse Gelsinger) in a clinical trial of gene therapy died, death is the body’s immune response of cytokine storm.

in 2000, doctors in the UK, France in moroni mice leukemia virus & other; Carrier & throughout; To supplement the normal gene in patients with severe combined immunodeficiency disease but because of the inserted genes inadvertently activated oncogenes, caused a number of patients with leukemia.

because of reflection and depressed for a long time of gene therapy in 2017 seems to have a new starting point. Two CAR – T gene therapy have been approved by the FDA. Madeleine’s personal fate and he brings a turning point in the field of gene therapy be? There is no answer yet.

on security, someone, someone still remains. Although adeno-associated virus is thought to have higher safety, but some worry that cannot predict side effects will still exist.

& other; So far, still there is no evidence to suggest that this treatment is potentially dangerous. Now should not be afraid. Throughout the &; Approved in the United States national institutes of health to participate in the project of Howard & middot; Kaufman (Howard Kaufman) said.

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