Republic of the Philippines

NUEVA VIZCAYA STATE UNIVERSITY

College of Arts and Sciences
Bambang, Nueva Vizcaya
WORKBOOK in SCIENCE, TECHNOLOGY AND SOCIETY
(GESTAS-2nd SEMESTER-2022-2023)
Name: Course & Year: Date: Score:

MODULE 10: GENE THERAPY

Activity No. 15: Case Study (25 points)
Read the case presented and answer the questions.

Case Study: 13-year-Old boy is First Person in US to Receive Newly Approved Gene Therapy for Blindness.

On Tuesday, a 13-year-old boy from New Jersey was at the center of medical history as he became the first
person in the US to receive an FDA-approved gene therapy for an inherited disease. The event marks the
beginning of a new era of medicine, one in which devastating genetic conditions that we are born with can be
simply edited out of our DNA with the help of modern biomedical technologies.

The therapy, Luxturna, from Spark Therapeutics, was approved by the FDA in December to treat a rare,
inherited form of blindness. Its price tag, set at $850,000-or $425,000 per eye made it the most expensive drug
in the US and sparked mass sticker-shock. But the therapy, which in high-profile clinical trials has allowed
patients to see the stars for the first times, also offered the almost miraculous possibility of giving sight to the
blind.

The therapy is intended to treat retinal diseases, including leber congenital amaurosis or retinisis pigmentosa,
cause by mutations in the RPE65 gene. The RPE65 gene produces an enzyme that helps the eye process
light. In these disorders, severe visual impairment begins often in infancy, and sometimes degrades over time.
Some people with a mutated copy of the gene can see during the day; others are legally blind. The drug works
by delivering a correct copy of the RPE65 gene to retinal cells, allowing the patient to produce the deficient
enzyme-and ,hopefully, restoring their vision.(Luxturna is considered by some to be the first “true gene therapy
”approved by the FDA, since other approved therapies, like those for blood cancers, involve removing a
patient’s cells from their body, modifying them externally, and then infusing them back into the body.)

Jack Hogan, the 13-year-old boy treated by doctors at Massachusetts Eye and Ear on Tuesday, began having
noticeable difficulty with his vision as a toddler. On Tuesday morning, he was wheeled into an operating room
at Massachusetts Eye and Ear, where doctors injected a modified virus into his eyes that would deliver the
correct RPE65 gene.

Doctors treated just one of Hogan’s eyes, with plans to treat the other next week. Time will tell if the treatment
worked, but the procedure so far has gone according plan.(Spark has set up a program to refund patients for
whom the therapy does not work, as part of the company’s campaign to justify the high price.)

Massachusetts Eye and Ear is one of eight medical centers certified to perform the gene therapy. Several
more procedures are already slated to take place across the country in the coming weeks.(Source:
https://gizmodo.com/doctors-perform-breakthrough-fda-approved-gene-therapy-1823954611 Posted on 22
March 2018)

Questions

1. What is the leber congenital amaurosis (LCA)?

2. What makes Luxturna a suitable treatment for LCA?

“In accordance with Section 185, Fair Use of Copyrighted Work of Republic Act 8293, the copyrighted works included in this material may be reproduced for educational
purposes only and not for commercial distribution,”
NVSU-FR-ICD-05-00 (081220) Page 1 of 5
 Republic of the Philippines
NUEVA VIZCAYA STATE UNIVERSITY
College of Arts and Sciences
Bambang, Nueva Vizcaya
WORKBOOK in SCIENCE, TECHNOLOGY AND SOCIETY
(GESTAS-2nd SEMESTER-2022-2023)
Name: Course & Year: Date: Score:

3. Based on article, identify the possible disadvantages of Luxturna?

4. What possible ethical and moral issues may arise from facilitating the gene therapy?

5. If you were the parents of the 13-year-old boy, would you allow your son to receive the gene therapy
treatment? Why or Why not?

“In accordance with Section 185, Fair Use of Copyrighted Work of Republic Act 8293, the copyrighted works included in this material may be reproduced for educational
purposes only and not for commercial distribution,”
NVSU-FR-ICD-05-00 (081220) Page 2 of 5
 Republic of the Philippines
NUEVA VIZCAYA STATE UNIVERSITY
College of Arts and Sciences
Bambang, Nueva Vizcaya
WORKBOOK in SCIENCE, TECHNOLOGY AND SOCIETY
(GESTAS-2nd SEMESTER-2022-2023)
Name: Course & Year: Date: Score:

MODULE 10: GENE THERAPY

Assignment No. 14. Gene Therapy Treatment

Get the names of people in the Philippines and abroad who will be treated by gene therapy. Indicate
the kind of treatment and their location.

NAMES TREATMENT LOCATION/

COUNTRY
1.

2.

3.

4.

5.

MODULE 10: GENE THERAPY

Evaluation No. 10 Gene Therapy

1-4 Challenges of gene therapy

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5-13 Advantages of Gene Therapy
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14-24 Disadvantages of Gene Therapy
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24.

“In accordance with Section 185, Fair Use of Copyrighted Work of Republic Act 8293, the copyrighted works included in this material may be reproduced for educational
purposes only and not for commercial distribution,”
NVSU-FR-ICD-05-00 (081220) Page 3 of 5