Gene Therapy Overview
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Questions and Answers

What is the primary change that occurs in a person's genetic composition due to gene therapy?

  • All cells within the body are altered.
  • Only the targeted cells change. (correct)
  • The genetic composition is completely modified.
  • The basic genetic composition changes significantly.
  • Which factor is NOT considered when determining eligibility for gene therapy?

  • Functionality of the affected areas.
  • Age of the patient.
  • Pre-existing antibodies.
  • Socioeconomic status. (correct)
  • What is the primary concern regarding long-term effects of gene therapy?

  • They tend to reverse with time.
  • Limited data available does not provide conclusive results. (correct)
  • They are universally positive.
  • They have been comprehensively studied.
  • Which of the following statements about gene therapy is FALSE?

    <p>Gene therapy treatments are proven to have clinical value.</p> Signup and view all the answers

    What potential consequence might receiving gene therapy have on future treatments?

    <p>It may restrict access to other types of therapies.</p> Signup and view all the answers

    What was a significant milestone achieved by MDA in gene therapy research?

    <p>First human gene therapy clinical trial for muscular dystrophy.</p> Signup and view all the answers

    Which neuromuscular diseases are mentioned as having gene therapies developed?

    <p>Spinal muscular atrophy and Duchenne muscular dystrophy.</p> Signup and view all the answers

    What is the amount invested by MDA in gene therapy for neuromuscular diseases over the past two decades?

    <p>$125M</p> Signup and view all the answers

    What is a primary concern regarding the long-term effects of gene therapy?

    <p>Long-term effectiveness and potential risks are not yet fully understood.</p> Signup and view all the answers

    Which factor does NOT influence the effectiveness of gene therapy?

    <p>Length of time the patient has been ill</p> Signup and view all the answers

    What role does a vector play in gene therapy?

    <p>It serves as a delivery mechanism for genetic materials.</p> Signup and view all the answers

    What can be a severe outcome of gene therapy?

    <p>Severe side effects or even death</p> Signup and view all the answers

    In what context is gene therapy considered irreversible?

    <p>It leads to lasting effects on the body's genetic instructions.</p> Signup and view all the answers

    What must patients and caregivers do before deciding on gene therapy?

    <p>Understand the potential long-term benefits and irreversible changes.</p> Signup and view all the answers

    What is a common misconception about gene therapy?

    <p>Gene therapy can provide a one-time cure with permanent results.</p> Signup and view all the answers

    How does gene therapy differ in its impact on individuals?

    <p>Outcomes can vary based on individual biological factors.</p> Signup and view all the answers

    What are the operating hours for MDA Gene Therapy Support staff?

    <p>Monday through Friday, 9 a.m. to 5 p.m. CT</p> Signup and view all the answers

    Which service is explicitly stated as available only in the US?

    <p>MDA services</p> Signup and view all the answers

    What is the expected response time for inquiries made to MDA Gene Therapy Support?

    <p>Within one to two business days</p> Signup and view all the answers

    Which professional is mentioned as a contributor to the document?

    <p>Barry J. Byrne, MD, PhD, University of Florida</p> Signup and view all the answers

    How can individuals reach out for assistance besides the phone number?

    <p>By sending an inquiry to the Resource Center email</p> Signup and view all the answers

    What is a critical disclaimer mentioned in the content?

    <p>Consultation with a healthcare team is essential for personalized information.</p> Signup and view all the answers

    Which statement reflects the outreach capability for those outside the US?

    <p>MDA can connect individuals to local muscular dystrophy groups.</p> Signup and view all the answers

    Which of the following is NOT a way to contact MDA?

    <p>By traditional mail</p> Signup and view all the answers

    Study Notes

    Gene Therapy

    • Gene therapy uses genetic materials to treat or prevent disease.
    • Genes contain instructions for our bodies to make proteins
    • Alterations in genes can impact how our bodies work
    • Gene therapy uses vectors to deliver genetic materials to cells
    • Gene therapy offers hope for individuals with neuromuscular disease
    • The long-term effects of gene therapy are unknown due to limited data

    Gene Therapy Facts

    • Gene therapy affects individuals differently and can cause side effects or even death
    • Gene therapies require months-long monitoring after infusion
    • Gene therapy is irreversible and can result in permanent changes to the body
    • A person's basic genetic composition remains unchanged

    Gene Therapy Eligibility

    • Not everyone is eligible for gene therapy
    • Factors such as age, pre-existing antibodies, and function can impact eligibility

    Gene Therapy Research

    • Over the past two decades, MDA has invested over $125M in gene therapy research
    • MDA investments have led to the development of clinical-ready gene therapies for diseases such as SMA and DMD
    • MDA-funded researchers conducted the first human gene therapy clinical trial for muscular dystrophy in 1999

    Gene Therapy Myths

    • Gene therapy is not a cure
    • Gene therapy is not simple
    • Gene therapy is not reversible
    • Gene therapy is not guaranteed to show beneficial results
    • Gene therapy treatments are not yet of proven clinical value
    • Gene therapy is not available to treat everyone with a neuromuscular disease
    • Any physician cannot prescribe gene therapy treatment safely

    MDA Involvement

    • MDA invested over $125M in gene therapy development over the past 20 years
    • MDA is working to facilitate access to gene therapy and provide support and education

    MDA Gene Therapy Support

    • Staff are available Monday through Friday
    • Answers to inquiries can be expected within one to two business days
    • MDA services are only available in the US

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    Description

    Explore the fundamentals of gene therapy through this quiz. Learn about how genetic materials are used to treat diseases, the implications of gene alterations, and the eligibility criteria for receiving such therapies. Understand the potential risks and ongoing research in the field of gene therapy.

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