Rami Major, PhD

genetics, bioethics, and sci pol 🧬✨

[Rami Major, PhD @doe_ra_mi]

Excited to share this paper from our multiyear, multidisciplinary project to understand the evolving governance of gene editing. One topic that kept bubbling up: the influence of the private sector on gene editing research🧵
https://t.co/clhTqX7vUC

[Rami Major, PhD @doe_ra_mi]

@MarkZylka @ASGCTherapy @aaas Thanks Mark, I’m looking forward to it!

[Mark Zylka @MarkZylka]

CRISPR/Cas works in mammalian cells, but it isn’t great. Why? We found it’s stuck to ribosomes in the cytosol. Not an ideal location for a genome editor.

People tacked on numerous nuclear localization signals (NLSs) to get Cas into the nucleus.

We asked a basic question: Why is it necessary to put so many NLSs on Cas9?

We figured out why in our latest study, led by @doe_ra_mi: https://t.co/auXNE6JL3o

[Rami Major, PhD @doe_ra_mi]

Excited to finally share this piece that has been many years in the making with one of my phenomenal dissertation advisors, Eric Juengst. We hope it can contribute to the ongoing conversations around prenatal gene editing applications in humans. Interested to hear your thoughts!

[AJHG @AJHGNews]

📢New Review from @doe_ra_mi & Juengst
📰Prenatal gene editing for neurodevelopmental diseases: Ethical considerations
https://t.co/1QuMQp5QB6

[Rami Major, PhD @doe_ra_mi]

@CBelmonteMateos @ASGCTherapy @MarkZylka @uncneuro Huge congrats Dr. Belmonte Mateos!!

[Rami Major, PhD @doe_ra_mi]

I hope this piece can add to the ongoing and incredibly important conversation about how to get rare gene therapies to the patients who need them in a way that makes sense for all involved. Interested to hear your thoughts!

[Rami Major, PhD @doe_ra_mi]

Excited to share the invited opinion piece I wrote with Zollie Yavarow out now in Molecular Therapy: “When promising therapies are out of reach: ethical responsibilities of stakeholders in gene therapy trials for rare disorders.”

https://t.co/zQ3Kg7MIge

[Rami Major, PhD @doe_ra_mi]

Out now! Our new pub on candidate criteria for gene editing governance:

https://t.co/femvla3osK

[Fyodor Urnov @UrnovFyodor]

Paraphrasing Gandhi:
"Be the edit you want to see in the world."
An actionable path to vigorous expansion of CRISPR cures in the next 5 years exists and is being actively pursued - eg by @somaticediting and @igisci x @DanaherCorp. Read below (it's free). 🙏 @CRISPRjournal.

[Rami Major, PhD @doe_ra_mi]

Wonder what gene editing stakeholders think about human heritable gene editing? Out now: “Human heritable genome editing and its governance: views of scientists and governance professionals”

Check it out here:
https://t.co/kFFmIk3aXk

[Rami Major, PhD @doe_ra_mi]

Last Department of Genetics Retreat in the books! Very fun to run through a quick preview of some of the work I’ll be sharing at my defense in a few weeks 👩‍🔬

[Rami Major, PhD @doe_ra_mi]

@jpflores_31 is everything you could hope for in a colleague and friend… not only does he do incredible things as a science advocate, he lifts others up with him as he does! Really enjoyed this chat with sci pol rockstar @AdrianaBankston and thanks JP for the invite to join!

[From where does it STEM? @wheredoesitstem]

Guest co-host Rami Major (@doe_ra_mi) and I interviewed Dr. @AdrianaBankston, a role model for many in the science policy & advocacy space! She will be the inaugural @aaas/@ASGCTherapy Congressional Policy Fellow starting this fall, and we are so excited to share her story 😊

[iScience journal @iScience_CP]

Online now: The Public-Private Research Ecosystem in the Genome Editing Era https://t.co/b6YkxaLDtB

[Rami Major, PhD @doe_ra_mi]

Bonus: if you’re interested in hearing what our interviewees had to say on the ethics of epigenetic editing, come find me at poster #1180 on Thursday at #ASGCT2024!

[Rami Major, PhD @doe_ra_mi]

Excited to share this paper from our multiyear, multidisciplinary project to understand the evolving governance of gene editing. One topic that kept bubbling up: the influence of the private sector on gene editing research🧵
https://t.co/clhTqX7vUC

[Rami Major, PhD @doe_ra_mi]

7/ I’m very grateful to be a part of a team asking big picture questions about gene editing that I feel like us bench science grad students don’t often get to consider about our field as a whole. Stay tuned for more of our work out soon ⏳