Medical Science 23

Advanced Genetics Part 2

April 3, 2024


Understand how different types of mutations behave, what a SNP is, and how n-Lorem uses all the available information to design an appropriate ASO for each patient.

Genetics can be intricate, no doubt. With millions of mutations present in every human, one might wonder why not everyone is impacted by pathogenic diseases. The Patient Empowerment Program aims to assist you in grasping the fundamental concepts of various mutations and how they function, paving the way for you to enhance your understanding of genetics. Delve into the diverse array of disease-causing mutations, their characteristics, and explore which mutations could potentially be addressed through ASO treatment in this concluding episode of Advanced Genetics.

On This Episode We Discuss:

  • The nature of a SNP
  • Pre-mature-m-RNA effects
  • Indels can disrupt the reading frame
  • Defining Alleles, Homozygous, Heterozygous, and Compound Heterozygous
  • The difference between whole exome and genome sequencing
  • How we, at n-Lorem, decide which patients are amendable to ASO treatments
  • How we design ASOs to take advantage of different post-RNA binding mechanisms
  • Mechanisms: Non-allele selective RNAse H1, Allele-selective RNAse H1, and Splicing ASOs

Watch on YouTube:

The host of the show is Dr. Stanley Crooke, a scientist, a physician, an entrepreneur and the father of antisense technology. Dr. Crooke is responsible for driving the development of antisense or ASO technology, an RNA-targeted technology responsible for the commercialization of three best- and first-in class medicines and more than 40 drugs in development. In 2020, Stan formed n-Lorem to use this powerful technology to develop experimental personalized ASO medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life.


Hosted by: Dr. Stan Crooke.
Videographer: Jon Magnuson of Mighty One Productions.
Producers: Colin Delaney, Kira Dineen, Jon Magnuson, Andrew Serrano and Amy Williford

More from n-Lorem:

See what else we are up to on Twitter, Instagram, Facebook, LinkedIn, YouTube and our website, If you enjoy this episode, please rate and review us, as it helps others find our podcast. Questions/inquiries can be sent to [email protected].

Listen to our next Intro to Medical Science episode:

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