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The rare disease that died before 10 was repaired by lentiviral gene therapy
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NEJM: The rare disease that died before the age of 10 was repaired by lentiviral gene therapy.
Hurler syndrome , also called mucopolysaccharidosis type IH (MPS-IH) , is a rare autosomal recessive genetic disease.
Due to genetic mutations, α-L-iduronidase (IDUA) is lacking, which in turn causes mucopolysaccharides to decompose and accumulate in the body.
It is the most common and severe type of mucopolysaccharidosis.
Children often have a period of normal development (a few weeks to 1 year) after birth, and then gradually develop physical and intellectual development disorders.
Most of them have significant symptoms at 16-18 months and usually die before the age of 10.
Allogeneic hematopoietic stem cell transplantation , is the standard treatment for Hurler syndrome, however, such treatment only partially cured and complications associated with high transplant-related mortality.
Recently, the ” New England Journal of Medicine ” (NEJM) published a study entitled: starved feeding and exhausting Stem- the Cell and Progenitor-Hurler Syndrome Gene Therapy for the clinical research papers.
Eight children with Hurler syndrome received autologous hematopoietic stem or progenitor cell therapy . These cells were transduced in vitro with a lentivirus encoding α-L- iduronidase . After treatment, the peripheral tissues and central The metabolism of the nervous system has been extensively corrected.
The eight children who received treatment lacked suitable donors for allogeneic hematopoietic stem cell transplantation , and their developmental quotient or IQ test scores were higher than 70, that is, none of them had moderate or severe cognitive impairment.
Under myeloablative conditions, children who were 1.9 ± 0.5 years old at the time of these treatments received autologous hematopoietic stem and progenitor cell (HSPC) transplants. These cells were derived from the patients themselves and were transduced in vitro with a lentivirus encoding α-L-iduronidase (IDUA) , allowing these cells to express α-L-iduronidase ( IDUA) .
The planned duration of this clinical trial is 5 years, and this paper is an interim report with a median follow-up time of 2.1 years. The interim report shows that the safety of this treatment is similar to other known autologous hematopoietic stem cell transplants.
All patients showed rapid and continuous implantation of genetic correction cells and produced active α-L-iduronidase (IDUA) within one month , and it continued. The level of mucopolysaccharides in urine dropped sharply. At 12 months after treatment, 4 of 5 evaluable children reached normal levels.
The presence of α-L-iduronidase (IDUA) could not be detected before in the cerebrospinal fluid of children, but it can be detected after treatment, and it is related to the local clearance of mucopolysaccharides.
More importantly, the treated children showed stable cognitive ability, stable motor skills corresponding to continuous motor development, improvement or stabilization of magnetic resonance imaging of the brain and spine, reduced joint stiffness, and compliance with World Health Organization growth Chart of normal growth and development.
8 children undergoing treatment
These results indicate that, for Hurler’s syndrome in children ‘s autologous hematopoietic stem or progenitor cells for gene therapy, metabolic children in peripheral tissues and the central nervous system has been widely corrected, also supported the further advance in the therapy clinic.
(source:internet, reference only)