Drs Peter Higgins and Judy Cho discuss recent success in sickle cell anemia and whether we're any closer to using intestinal gene therapy in Crohn's disease. Relevant disclosures can be found with the episode show notes on Medscape.com (https://www.medscape.com/viewarticle/967621). The topics and discussions are planned, produced, and reviewed independently of advertiser. This podcast is intended only for US healthcare professionals.

Resources

Sickle Cell Anemia https://emedicine.medscape.com/article/205926-overview

Crohn's Disease https://emedicine.medscape.com/article/172940-overview

Inflammatory Bowel Disease https://emedicine.medscape.com/article/179037-overview

CRISPR-Cas systems: Overview, Innovations and Applications in Human Disease Research and Gene Therapy https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7508700/

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia https://www.nejm.org/doi/full/10.1056/NEJMoa2031054

Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease https://www.nejm.org/doi/10.1056/NEJMoa2117175

Causes of Crohn's disease https://medlineplus.gov/genetics/condition/crohn-disease/#causes

IL-10R Polymorphisms Are Associated With Very-Early-Onset Ulcerative Colitis https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3744177/

Characterization of Crohn Disease In X-Linked Inhibitor of Apoptosis-Deficient Male Patients and Female Symptomatic Carriers https://pubmed.ncbi.nlm.nih.gov/24942515/

A Frameshift Mutation in NOD2 Associated With Susceptibility to Crohn's Disease https://www.nature.com/articles/35079114

Extended Haplotype Association Study in Crohn's Disease Identifies a Novel, Ashkenazi Jewish-Specific Missense Mutation in the NF-κB Pathway Gene, HEATR3 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3785105/

Autologous Stem Cell Transplant for Crohn's Disease https://www.mountsinai.org/clinical-trials/autologous-stem-cell-transplant-for-crohns-disease

Stem-Cell Transplantation for Crohn's Disease: Same Authors, Different Conclusions? https://www.thelancet.com/journals/langas/article/PIIS2468-1253(17)30076-6/fulltext#%20

A Myeloid-Stromal Niche and gp130 Rescue in NOD2-Driven Crohn's Disease https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8284870/

Expression of NOD2 in Paneth Cells: A Possible Link to Crohn's Ileitis https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1773866/

Role of ATG16L, NOD2 and IL23R in Crohn's Disease Pathogenesis https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3270503/