Week 1 HW: Principles and Practices

1) Describe a biological engineering application or tool you want to develop and why.

I’d like to explore the development of CRISPR/Cas9 applications for genetic neurological disorders such as Parkinson’s disease and Amyotrophic Lateral Sclerosis which I inspired from Lecture 1, Ricky Cordova talk about ovarian cancer. My interest in this field stems from my background in physical therapy as I’m currently a junior student. Through my clinical experience in rehabilitation programs, I’ve observed that treatment primarily focuses on improving muscle tone, motor function and neural recovery to maximize patients’ functional abilities. I believe that integrating gene therapeutic approaches with targeted rehabilitation programs could repair the underlying causes of these disorders while enhancing functional recovery and ultimately improving patient outcomes and independence.

2) Describe one or more governance/policy goals related to ensuring that this application or tool contributes to an “ethical” future, like ensuring non-malfeasance (preventing harm). Break big goals down into two or more specific sub-goals.

1. Make sure the treatment is proven safe before using it on patients, The therapy must be tested carefully in labs and clinical trials, Official health authorities must approve it before doctors can use it.

2. Monitor patients for a long time after treatment As gene editing can have long-term effects, patients should be followed and checked regularly. Doctors should track changes in brain function, movement, and overall health.

3) Describe at least three different potential governance “actions” by considering the four aspects below (Purpose, Design, Assumptions, Risks of Failure & “Success”).

1. Purpose: Integrating gene-based therapeutic approaches alongside with the rehabilitation program

2. Design: ASGCT, WHO and and national health regulatory authorities can support and approve the clinical use of gene therapy, These organizations would help establish safety guidelines, clinical trials and require long-term follow-up studies to ensure treatment safety and effectiveness

3. Assumptions: Integrating gene-based therapy with rehabilitation programs may be uncertain because some patients may not regain sufficient neurological function to fully participate in rehabilitation, The approach assumes that gene therapy will improve neural function enough for rehabilitation to enhance recovery

4. Risks of Failure & “Success”: If the gene therapy doesn’t improve the patient’s condition enough, the rehabilitation program may not lead to meaningful recovery

4) Next, score (from 1-3 with, 1 as the best, or n/a) each of your governance actions against your rubric of policy goals. The following is one framework but feel free to make your own:

5) Drawing upon this scoring, describe which governance option, or combination of options, you would prioritize, and why. Outline any trade-offs you considered as well as assumptions and uncertainties.

Based on the evaluation, I would prioritize strict safety approval and long-term monitoring of CRISPR therapies. These actions best protect patients by ensuring treatments are safe before use and by observing long-term effects. This recommendation is directed toward national health authorities, international health organizations like WHO. A trade-off is that strong regulation may slow innovation and delay access to treatment but safety should be the priority because gene editing can cause permanent biological changes. This approach assumes regulatory systems are effective and that long-term outcomes can be monitored although uncertainty remains about patient response and long-term effects.

Week 1 Lab: Pipetting

Individual Final Project

Group Final Project