Couverture de Patient Empowerment Program: A Rare Disease Podcast

Patient Empowerment Program: A Rare Disease Podcast

Patient Empowerment Program: A Rare Disease Podcast

De : n-Lorem Foundation (Dr. Stan Crooke Amy Williford Kim Butler Andrew Serrano Jon Magnuson and Kira Dineen)
Écouter gratuitement

Join the nano-rare disease community! Interviews features leading physicians, scientists, biotech experts, and patient advocates. Lessons teach core concepts about drugs. Our host Dr. Crooke has led the creation of antisense technology and his foundation, n-Lorem, is using this powerful technology to discover, develop, and provide personalized experimental antisense oligonucleotide medicines to nano-rare patients for free, for life. n-Lorem is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat patients (less than 30 patients) that are the result of a single genetic defect unique to only one or very few individuals. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. n-Lorem was founded by Dr. Stan Crooke, who founded IONIS Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. The podcast is produced by n-Lorem Foundation and hosted by Dr. Stanley T. Crroke, who is the Founder, CEO and Chairman. Our videographer is Jon Magnuson. Our producers are Kira Dineen, Jon Magnuson, Kim Butler, and Amy Williford. To learn more about n-Lorem, visit nlorem.org. Contact us at podcast@nlorem.org.Copyright 2024 n-Lorem Foundation Hygiène et vie saine Maladie et pathologies physiques Science
Épisodes
  • Miracles of Science #3: RNA World Part 3
    Jun 3 2026

    In this episode of the n-Lorem Patient Empowerment Program Podcast, host Stan Crooke explores the fascinating world of RNA biology and explains how different types of RNA work together inside cells. He breaks down the roles of ribosomal RNA, messenger RNA, transfer RNA, and several regulatory and processing RNAs, showing how they help convert genetic information into proteins and control cellular function. The episode also highlights how advances in RNA science are helping the n-Lorem Foundation develop individualized treatments for nano-rare patients and their families.

    • 0:00 – Introduction to the mission of n-Lorem and hope for nano-rare patients and families.
    • 0:51 – Stan Crooke introduces the podcast and explains n-Lorem’s groundbreaking treatment model.
    • 1:12 – Overview of the RNA world and why different RNA types are essential to biology.
    • 1:48 – Introduction to translational RNAs and their role in making proteins.
    • 2:16 – Explanation of ribosomes and how they translate genetic code into proteins.
    • 5:12 – Discussion of pre-ribosomal RNA processing and the role of the nucleolus.
    • 7:48 – Ribosomes are compared to the Enigma machine for decoding biological information.
    • 9:29 – Overview of transfer RNAs (tRNAs), codons, and amino acid delivery.
    • 12:23 – Why decoding the genetic code was a landmark scientific breakthrough.
    • 14:18 – How tRNAs mature through RNA processing and splicing mechanisms.
    • 16:03 – Summary of how mRNA, rRNA, and tRNA work together during translation.
    • 18:37 – Introduction to processing RNAs and spliceosome machinery.
    • 21:15 – Explanation of U4 RNA and its role in RNA quality control and disease.
    • 23:00 – Overview of snoRNAs and their role in ribosomal RNA maturation.
    • 24:06 – Discussion of Cajal body RNAs and RNA modification systems.
    • 25:01 – Introduction to regulatory non-coding RNAs and antisense RNAs.
    • 27:50 – Overview of circular RNAs and their potential regulatory functions.
    • 28:51 – Explanation of microRNAs and how they regulate protein production.
    • 29:59 – Closing thoughts on regulatory RNAs and cellular control systems.
    Afficher plus Afficher moins
    36 min
  • Miracles of Science #3: RNA World Part 2
    May 27 2026

    In Part 2 of the n-Lorem Patient Empowerment Program podcast Miracles of Science series on RNA, Dr. Stan Crooke further explores the fascinating “RNA world,” explaining how RNA molecules are processed, protected, modified, and used to manage cellular function. The episode breaks down concepts like pre-RNAs, poly-A tails, 5’ caps, RNA splicing, and the multiple “codes” that make RNA dynamic and information-rich — foundational science that helps power antisense therapies for nano-rare patients and helps make the RNA world a true "Miracle of Science," allowing n-Lorem to fulfil its mission.

    • Shownotes:
    • 0:00 — Introduction to n-Lorem Overview of the n-Lorem Foundation mission and support for nano-rare patients and families.
    • 0:15 — Podcast & Sponsor Introduction Dr. Stan Crooke introduces the podcast and acknowledges sponsor ChemGenes.
    • 1:17 — Entering the “RNA World” Dr. Crooke introduces RNA biology and explains why RNA science is foundational to antisense therapies.
    • 1:31 — What Are Pre-RNAs? Explanation of immature “pre-RNAs” and how they contain instructions for cellular processing and transport.
    • 3:31 — RNA Processing & Adding Nucleotides Discussion of how RNAs are trimmed and modified during maturation.
    • 4:03 — Poly-A Tails Explained What poly-A tails are, why they are added to messenger RNA, and how they protect RNA molecules.
    • 7:07 — Protecting RNA from Degradation How cells chemically protect both ends of RNA molecules.
    • 9:00 — The 5’ Cap Introduction to RNA “caps” and their role in identifying and stabilizing messenger RNA.
    • 11:02 — RNA Splicing How cells remove unnecessary RNA segments (introns) and reconnect useful coding regions.
    • 14:06 — RNA Structural Codes How RNA folds into structures and why shape is important for function.
    • 16:09 — Chemical Modification Codes Overview of RNA chemical modifications and how they add another layer of cellular regulation.
    • 18:22 — RNA as Cellular Management Dr. Crooke compares RNA molecules to executives and middle managers directing cellular operations.
    • 19:12 — RNA & Antisense Technology Why understanding RNA biology is critical for developing antisense therapies for nano-rare diseases.
    • 22:08 — DNA vs. RNA Analogy DNA as the “king” and RNA as the active workforce managing the affairs of the cell.
    • 24:10 — The Dynamic Nature of RNA How RNA constantly changes structure, interactions, and function over time.
    • 26:14 — Closing Thoughts Final overview of RNA versatility and its role in cellular communication and regulation.
    • 26:54 — About n-Lorem Information about n-Lorem’s mission to provide personalized experimental treatments free for life to nano-rare patients.
    Afficher plus Afficher moins
    28 min
  • Miracles of Science #3: RNA World Part 1
    May 20 2026

    In this episode of the n-Lorem Patient Empowerment Podcast, Stan Crooke explores the “RNA World” — the groundbreaking field of RNA biology that helped make modern genetic medicine possible. Drawing from personal experience, he shares the scientific battles surrounding early RNA discoveries, the development of antisense technology, and how decades of persistence ultimately led to life-changing therapies for nano-rare patients. Along the way, Stan explains how evolution, molecular biology, and information systems inside cells shape human health and the future of medicine.

    00:00 – Introduction & Sponsor Message Stan introduces the episode and explains the importance of the RNA World to nano-rare medicine.

    02:15 – Why RNA Science Changed Everything How RNA discoveries became foundational to modern genetic therapies.

    05:10 – The Scientific Wars Over Small Nuclear RNAs The fierce debates among scientists about whether snRNAs were real or experimental artifacts.

    09:40 – What Science Is Really Like Behind the Scenes Stan discusses the emotional and competitive nature of scientific discovery.

    13:00 – Antisense Technology & the Road to Spinraza How decades of RNA research led to breakthrough treatments for spinal muscular atrophy.

    17:30 – Lessons From Evolution Why evolution reuses successful molecular strategies to create complex life.

    22:15 – Families of Genes, Proteins & Molecular Efficiency How biological systems adapt and customize molecular functions.

    26:00 – Life as an Information System Stan explains DNA, RNA, proteins, and cells through the lens of information theory.

    29:00 – Final Thoughts: Humanity Wins Through Science Why scientific perseverance ultimately benefits patients and society.

    Afficher plus Afficher moins
    34 min
adbl_web_anon_alc_button_suppression_t1
Aucun commentaire pour le moment