1. Gene Therapy and Viral Vector Lecture 4

2. Cystic Fibrosis Average life span:25-30 years

3. Conventional Treatment the use of physiotherapy, antibiotics and pancreatic supplements. Many patients require treatment four times daily, including a considerable time spent on physiotherapy. This combination of treatment has helped increase life expectancy considerably to the current median of approximately 30 years of age. Despite small further improvements, more recently it has become apparent that there is a need for a more effective and convenient therapy. The identification of the gene responsible for CF (the cystic fibrosis transmembrane conductance regulator or CFTR protein) in 1989, was responsible for the advent of potential new treatments for CF. Gene therapy, the transfer of a normal copy of the CFTR gene into the lungs of CF patients, was proposed as an attractive new option.

4. Cystic Fibrosis, a case study http://learn.genetics.utah.edu/content/gen etherapy/casestudy/

5. News of Gene therapy in CF http://www.bbc.com/news/science- environment-32932922 http://www.bbc.com/news/science- environment-32932922 http://www.nhs.uk/news/2015/07July/Page s/Gene-therapy-breakthrough-for-cystic- fibrosis.aspx http://www.nhs.uk/news/2015/07July/Page s/Gene-therapy-breakthrough-for-cystic- fibrosis.aspx 3 July 2015

6. Assignment Group 1: AIDS/HIV Group2: Arthritis Group 3: Hemophilia Group 4: Hypertension Group 5: Diabetes Group 6: Hepatitis Group 7: Cancer (Any type) Group 8: Cancer (Any type)

7. Topics to be covered in the assignment Introduction of the disease or disorder Type(s) of mutations involved Conventional treatment Type of vector The procedure of gene therapy History of clinical trials At which phase of clinical trial the treatment is undergoing? Is it commercially available? Deadline : 30 Oct

8. Personal Statement ‘Every viable candidate for the most competitive academic fellowships has a high GPA and stellar recommendations. What distinguishes the top candidates (the ones who are invited for interviews) from the others is the quality of the personal statement. Your personal statement should tell a story about you that makes the selection committee members want to meet you. It should demonstrate your humanity as well as your destiny for greatness. Selection committees are looking for candidates who have already shown that they are capable of doing great things. They are also attracted to candidates who care about people, who want to “fight the good fight” and who can inspire others around them to join in those efforts. This can mean making important contributions in one's chosen field of endeavor, or making a difference in the world in some substantial way. Recent fellowship winners represent a wide variety of academic backgrounds and career interests.’

9. How to write it? Think of your characteristics or actions that make you distinctive. How would your friends describe what's important about you to someone who doesn't know you? Try writing a story about an incident from your life that illustrates one of these characteristics. Think of one of the most significant learning experiences in your life -- an Aha! moment -- when you finally understood something for the first time. Write about this experience and relate it to your development and your aspirations. What do you care about most deeply? What matters to you? How have you spent your time in the past few years toward working to further this passion or dream? What are your plans for fulfilling your dreams?

10. Sample of Personal Statement https://www.e- education.psu.edu/files/writingpersonalsta tementsonline/file/Fulbright.pdf https://www.e- education.psu.edu/files/writingpersonalsta tementsonline/file/Fulbright.pdf