What is gene therapy?
Current treatments for mucolipidosis mainly focus on symptomatic relief. However, with the advancement in genetic engineering and molecular biology, gene therapy has emerged as a promising avenue.
Gene Therapy: The Basics
Gene therapy seeks to treat genetic disorders by introducing, altering, or replacing a DNA sequence within the cell. For Mucolipidosis, the hope lies in targeting the faulty gene responsible for the malfunctioning process and replacing or repairing it.
Gene replacement involves introducing a healthy copy of the affected gene into a patient's cells. Special viruses that have been modified to carry human DNA (known as vectors), transport this healthy gene into cells. Once inside, the hope is that the cell will start to produce the functional enzyme, alleviating the disease's effects.
AAV9: A Tiny Hero in Gene Therapy
Imagine you want to send a letter to a friend who lives in a locked house. You need a special mail carrier who can slip the letter inside without causing any disturbance. In the world of gene therapy, AAV9 acts like this special mail carrier, delivering vital healing messages into our cells.
AAV9 is a friendly visitor that our body doesn't mind having around. Our immune system, which usually chases away foreign entities, doesn't get too upset with AAV9. Once AAV9 drops off its healing message inside our cells, that message can keep working for a long time.
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