TRANSCRIPT: https://primaryimmune.org/sites/default/files/TRANSCRIPT%20Discussing%20Nobel%20Prize.pdf
Dr. Troy Torgerson (Allen Institute for Immunology, Immune Deficiency Foundation Medical Advisory Committee) and Katherine Lontok (Director of Science and Policy Communications, Immune Deficiency Foundation) discuss the Nobel Prize-winning research of Mary Brunkow, Fred Ramsdell and Shimon Sakaguchi.
Find Dr. Torgerson on LinkedIn: https://www.linkedin.com/in/troy-torgerson-44b6207/
Your support of the Immune Deficiency Foundation helps us spread the word about primary immunodeficiency and those who live with it. To support our work, please donate at primaryimmune.org/donate.
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TRANSCRIPT: https://primaryimmune.org/sites/default/files/TRANSCRIPT%20Discussing%20Nobel%20Prize.pdf
Dr. Troy Torgerson (Allen Institute for Immunology, Immune Deficiency Foundation Medical Advisory Committee) and Katherine Lontok (Director of Science and Policy Communications, Immune Deficiency Foundation) discuss the Nobel Prize-winning research of Mary Brunkow, Fred Ramsdell and Shimon Sakaguchi.
Find Dr. Torgerson on LinkedIn: https://www.linkedin.com/in/troy-torgerson-44b6207/
Your support of the Immune Deficiency Foundation helps us spread the word about primary immunodeficiency and those who live with it. To support our work, please donate at primaryimmune.org/donate.
The information, terminology, and opinions presented in this forum do not necessarily reflect the views of IDF, its Board of Trustees, sponsors, or donors.
Decoding PI: The science of gene therapy and gene editing
Immune Deficiency Foundation
1 hour 14 minutes 45 seconds
7 months ago
Decoding PI: The science of gene therapy and gene editing
Transcript: https://primaryimmune.org/sites/default/files/01PROOF%20transcription%20gene%20editing%20webinar.pdf
View a PDF of this presentation: https://primaryimmune.org/sites/default/files/FOR%20WEB%20-%20Gene%20editing.pdf
Susan Prockop, MD of Boston Children's Hospital and Harvard Medical School provides an overview and recent updates to gene therapy research for primary immunodeficiency.
To learn more, volunteer, or donate, visit primaryimmune.org/donate.
The information, terminology, and opinions presented in this forum do not necessarily reflect the views of IDF, its Board of Trustees, sponsors, or donors.
LINKS:
SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection: https://clinicaltrials.gov/study/NCT03217617?term=NCT03217617&rank=1
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 with Low Dose Targeted Busulfan Conditioning: https://clinicaltrials.gov/study/NCT03311503?term=NCT03311503&rank=1
Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID): https://clinicaltrials.gov/study/NCT01306019?term=NCT01306019&rank=1
Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome: https://clinicaltrials.gov/study/NCT01410825?term=NCT01410825&rank=1
Gene Therapy for WAS: https://clinicaltrials.gov/study/NCT01347346?term=NCT01347346&limit=10&rank=1
Gene Therapy for Wiskott-Aldrich Syndrome (WAS): https://clinicaltrials.gov/study/NCT01347242?term=NCT01347242&limit=10&rank=1
Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease: https://clinicaltrials.gov/study/NCT02234934?term=NCT02234934&limit=10&rank=1
Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD) (CGD): https://clinicaltrials.gov/study/NCT01855685?term=NCT01855685&limit=10&rank=1
pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD): https://clinicaltrials.gov/study/NCT06253507?term=NCT06253507&limit=10&rank=1
Lentiviral Gene Therapy for p47 AR-CGD: https://clinicaltrials.gov/study/NCT05207657?term=NCT05207657&limit=10&rank=1
Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease: https://clinicaltrials.gov/study/NCT06325709?term=NCT06325709&limit=10&rank=1
A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants with P47phox Autosomal Recessive Chronic Granulomatous Disease (CGD): https://clinicaltrials.gov/study/NCT06559176?term=NCT06559176&limit=10&rank=1
Immune Deficiency Foundation
TRANSCRIPT: https://primaryimmune.org/sites/default/files/TRANSCRIPT%20Discussing%20Nobel%20Prize.pdf
Dr. Troy Torgerson (Allen Institute for Immunology, Immune Deficiency Foundation Medical Advisory Committee) and Katherine Lontok (Director of Science and Policy Communications, Immune Deficiency Foundation) discuss the Nobel Prize-winning research of Mary Brunkow, Fred Ramsdell and Shimon Sakaguchi.
Find Dr. Torgerson on LinkedIn: https://www.linkedin.com/in/troy-torgerson-44b6207/
Your support of the Immune Deficiency Foundation helps us spread the word about primary immunodeficiency and those who live with it. To support our work, please donate at primaryimmune.org/donate.
The information, terminology, and opinions presented in this forum do not necessarily reflect the views of IDF, its Board of Trustees, sponsors, or donors.