Human Gene Therapy For Rare Disease
The idea behind gene therapy is simple : a person is born with a version of a gene that causes a disease, so you deliver a new gene to resolve the issue. More than 80% of rare diseases have a known genetic component. At Pfizer Rare Disease, we are exploring the potential of gene therapy to treat these rare genetic diseases through potentially life-changing innovations, trusted partnerships, and relentless passion. it has become clear that genetics may not have to determine the future of a person living with a rare disease. Gene therapy is a potentially transformative treatment approach that, if successful, may help reduce the burden of ongoing treatments for nearly 320 million people worldwide who are affected by rare genetic diseases. If we are successful, imagine the possibilities.
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Gene therapy is used to cures for many diseases and types of medical treatment. Many diseases such as, cancers, neurological disorders, cardiovascular disease, infectious diseases and many more rare diseases. Gene therapy encompasses the manipulation of genes to fight or prevent diseases. The therapy is introduces a good gene into a person who has a disease caused by a bad gene. Many of treatments are include, biosimilars, vaccines, complex generics, gene therapies, immuno-therapies and novel drugs.
Gene therapy in rare diseases generally happens in one of two ways: the vector may be injected directly into the affected tissue or the cells could be modified at a genetic level and reinserted in the body. The former is common when an organ is impacted. This approach helps minimize undesirable effects and promotes the uptake of gene therapy. The latter is seen more in haematological disorders. There is a third method that involves transplanting cells into the body, such as seeking to treat an eye disorder by transplanting retinal cells or using CAR T-cells (chimeric antigen receptor T-cells) to target tumour antigens. The cell source determines how gene or cell therapies are categorized. In some cases, the therapy is patient-specific while others are designated “off-the-shelf.”
A rare disease is defined as one that affects fewer than 200,000 people across a broad range of possible disorders. Chronic genetic diseases are commonly classified as rare.Among numerous possibilities, rare diseases may result from bacterial or viral infections, allergies, chromosome disorders, degenerative and proliferative causes, affecting any body organ. Rare diseases may be chronic or incurable, although many short-term medical conditions are also rare diseases.
There are currently about 5 million patients, which will rise because the population is aging. Then, if we are trying to treat thousands of patients, that’s when it’s hard. As a field, we have to start preparing for it today or we’re going to be in a worse situation
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