Rhys' story - a Jeans for Genes Day film

Summary

This video highlights the emotional journey of families affected by genetic disorders and the groundbreaking work being done to provide hope through gene therapy. The story of Reese Evans, a child born with severe combined immunodeficiency, showcases the potential of gene therapy in treating genetic conditions. With the support of medical research and the Genes for Genes appeal, scientists are using genetic information to develop new treatments, offering a life-changing chance for those with rare genetic diseases. Though gene therapy is still in its early stages, it promises a brighter future for many children like Reese.

Takeaways

• 😀 Genetic conditions affect 1 in 33 babies born in the UK today, and there are over 4,000 known genetic disorders.
• 😀 Many genetic conditions currently have no cure, but ongoing research is crucial for finding solutions.
• 😀 The Genes for Genes appeal raises funds for medical research and supports families coping with genetic conditions.
• 😀 The appeal helps provide specialist medical care and support for families, funding the activities of several charities each year.
• 😀 The story of Reese Evans highlights the hope that comes from genetic research and gene therapy.
• 😀 At 4 months old, Reese was hospitalized with an incurable infection, and doctors feared for his life.
• 😀 Reese’s condition was a severe combined immunodeficiency, meaning his immune system was almost non-functional.
• 😀 After 21 days of uncertainty, Reese was referred to a gene therapy team at Great Ormond Street Hospital for treatment.
• 😀 The gene therapy used in Reese’s case involved modifying a virus to deliver a working copy of the defective gene into his cells.
• 😀 Gene therapy is still in its early stages, and while it offers hope for the future, finding cures for all genetic conditions will take time and funding.

Q & A

What is the main focus of the Genes for Genes appeal?

The Genes for Genes appeal focuses on raising funds for research into genetic disorders and providing support for families affected by these conditions.

How many genetic disorders are there, and how many babies are affected?

There are over 4,000 genetic disorders, and one in 33 babies is born with a genetic condition that can affect their health.

What is the main goal of the research funded by Genes for Genes?

The goal of the research is to develop treatments and ultimately cures for genetic disorders, improving the lives of those affected.

How does Genes for Genes support families affected by genetic conditions?

Genes for Genes helps families by providing specialist medical care and support services, as well as funding research into better treatments.

What kind of treatments is the Genes for Genes appeal funding?

The appeal is funding research into gene therapy, a pioneering treatment that aims to correct defective genes in patients with genetic disorders.

What is the significance of Reese Evans' story?

Reese Evans' story highlights the potential of gene therapy as a treatment for severe genetic conditions. Despite facing a life-threatening condition, Reese was saved through a combination of medical care and gene therapy.

What was the condition Reese Evans suffered from?

Reese Evans suffered from severe combined immunodeficiency (SCID), a genetic disorder that left him with no functioning immune system.

How was Reese Evans' condition diagnosed and treated?

Reese's condition was diagnosed after extensive tests revealed he had no immune system. He was treated with gene therapy, which involved introducing a working copy of his defective gene to help restore his immune system.

How does gene therapy work in treating genetic disorders?

Gene therapy works by modifying a patient's own cells, introducing a correct or working copy of the defective gene. This is often done using harmless modified viruses to deliver the new gene into the patient's cells.

Is gene therapy a proven cure for all genetic disorders?

No, gene therapy is still in its early stages, and while it offers hope for some conditions, it is not yet a cure for all genetic disorders. Each condition requires a unique approach.