Why in News
A treatment protocol that combines autologous stem cell transplants with gene therapy shows signs of correcting skeletal deformities in eight children with Hurler syndrome.
A rare disease that stunts skeletal system growth.
Promising results from a phase 1 trial suggest that similar strategies may counteract one of the debilitating complications of this rare disorder, which has no cure.
Patients who received autologous stem cells containing the corrected gene displayed close-to-normal skeletal growth patterns.
Hurler syndrome
Hurler syndrome, also known as mucopolysaccharidosis type I (MPS I).
It is a rare genetic disorder.
It's a lysosomal storage disease, which means the body is missing or has a deficiency in a specific enzyme needed to break down complex sugar molecules called glycosaminoglycans (GAGs).
What is stem cell
They can divide and copy themselves over a long period of time, replenishing their pool.
They have the potential to develop into many different types of specialized cells, like muscle cells, nerve cells, or blood cells.
This characteristic is what makes them so valuable.
There are two main types of stem cells:
Embryonic stem cells (ESCs): These are pluripotent, meaning they have the ability to develop into any cell type in the human body.
They are derived from the inner cell mass of a blastocyst, an early-stage embryo.
Due to ethical considerations, research on embryonic stem cells is limited in many countries.
Adult stem cells (ASCs): These are found in various tissues throughout the body, like bone marrow, blood, or fat.
They are multipotent, meaning they can develop into a limited number of cell types specific to their tissue of origin.
They can be a valuable source of cells for regenerative medicine.
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