Lentiviral gene therapy cures severe combined immunodeficiency
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NEJM: Lentiviral gene therapy cures two types of severe combined immunodeficiency
Lentiviral gene therapy cures severe combined immunodeficiency. On September 21, 1971, a boy named David Phillip Vetter was born. However, unfortunately, he suffered from an extremely rare genetic defect disease-severe combined immunodeficiency Illness (SCID for short), from the moment he was born, he lived in a sterile transparent plastic isolation hood, so David is also called “Bubble Boy” (Bubble Boy).
On February 22, 1984, David, who had struggled with illness and loneliness for 12 and a half years, enjoyed the dignity and freedom of life and passed away quietly. Before his death, David’s mother finally touched him, the first and last time.
Severe combined immunodeficiency disease (SCID), there are two main types of the disease, one is caused by adenosine deaminase (ADA) deficiency, referred to as ADA-SCID, which is an autosomal recessive genetic disease, and the other is caused by interleukin 2 The receptor (IL-2R) defect is caused by a recessive genetic disease with X chromosome.
Severe combined immunodeficiency disease (SCID) is very rare and very serious, with an incidence of only one in 100,000. People with SCID do not have any immune system in their body, so they do not have any ability to resist bacteria and viruses. For patients, the outside world is full of fatal threats, and even a loving kiss or hug from the mother may bring terrible consequences to them, often due to infection soon after birth.
On May 11, 2021, the New England Journal of Medicine (NEJM) published a gene therapy clinical trial study titled Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
The research team used the patient font CD34+ hematopoietic stem and progenitor cells (HSPC) to transduce the correct adenosine deaminase (ADA) gene through self-inactivating lentivirus in vitro. These cells, which have been genetically repaired by the lentivirus, are then returned to the patient’s body.
The gene therapy was developed by Orchard Therapeutics, which was established in 2015 to treat rare genetic diseases through gene therapy.
The gene therapy has a total of 50 patients, including 30 in the United States and 20 in the United Kingdom. The United States patients used fresh lentiviral gene repair HSPC cell preparations and cryopreserved cell preparations, and followed up for 24 months. British patients all used fresh HSPC cell preparations with lentiviral gene repair and followed up for 36 months.
The clinical results show that the lentiviral gene therapy for Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) has a significant effect. At the 24-month and 36-month follow-up, the overall survival rate of all 50 treated patients was 100%.
In the US study, 29 out of 30 patients had persistent lentiviral gene repairing HSPC cells. In the UK study, 19 out of 20 patients had persistent lentiviral gene repairing HSPC cells, and the glands of these patients The level of glycoside deaminase (ADA) activity returned to normal. And their immune system recovered well. 90% of American patients and 100% of British patients stopped immunoglobulin replacement therapy at the 24th and 36th months.
In addition, none of the patients found any signs of monoclonal amplification, leukocyte proliferative complications, or replication-type lentiviruses, and no autoimmune or graft-versus-host disease events occurred. Most of the adverse events were low-grade.
Prior to this, on April 18, 2019, the New England Journal of Medicine (NEJM) published a research paper from the St. Jude Children’s Research Hospital in the United States. The research team used the lentiviral gene therapy developed by Mustang Bio. Combined with low-dose busulfan, 8 babies with SCID-X1 were successfully treated.
These two gene therapy clinical trials indicate that the two main types of severe combined immunodeficiency (SCID) have been overcome by lentiviral gene therapy.
(source:internet, reference only)
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