Timeline of Gene

Theraphy
Presented by Group 5
 Timeline of Gene Theraphy
• 1977 Scientists were able to deliver target genes into mammalian cells

• 1990's Sickle cell disease successfully treated in mice with gene therapy. First human trial
experiment with gene therapy First journal published (Human Gene Therapy) • First death reported
due to gene therapy

• 2000s Number of diseases with successful gene therapy increases. First gene therapy drug
introduced by China

• 2010+ Focus shifts from single gene defects to multigene defects

• 2011 Medical community accepts gene therapy to successfully treat HIV

• 2017 Gene therapy based drugs approved

Successfully Treated Diseases with Gene Therapy

Gene Theraphy

• Cystic fibrosis
• Haemophilia
• Thalassemia
• Sickle Cell Anaemia Familial
Hypercholesterolemia
• Severe Combined Immunodeficiency
 Gene Theraphy
The majority of gene therapy trials are being conducted in the United States
and Europe.
Advantages :

• Can be used to "silence" a disease before its onset (Ex: HIV in AIDS)
• Potential to eliminate and prevent hereditary diseases.
• The "Last Chance" or "Last Hope" therapy.
• Achieving pharmacological effects (Ex: Making cancer cells susceptible to
anticancer drugs.)
 Gene Theraphy
The majority of gene therapy trials are being conducted in the United States and
Europe.
Disadvantages :
• Short-lived nature (Repeated therapy)
• Severe Adverse Effects (Ex: Viral vectors may trigger an extreme immune
response).
• Viral vectors could recover the ability to cause disease once inside the patient.
• Difficulty to effectively treat multigenic disorders (Ex: Diabetes and Heart
Disease)
• Ethical and Social Considerations High cost makes this "promising"
therapy available only to the wealthy.
Ethical Issues Surrounding Gene Therapy

• How can "good" and "bad" uses of gene therapy be distinguished? • Who
decides which traits are normal and which constitute a disability or disorder?
• Will the high costs of gene therapy make it available only to the wealthy?
• Could the widespread use of gene therapy make society less accepting of people
who are different? • Should people be allowed to use gene therapy to enhance
basic human traits such as height, intelliger athletic ability?
-Gene therapy is still new ad experimental.