Gene Editing
14 videos · last 6 months shuffled daily, then older videos newest first
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Walter Koroshetz on the State of Epilepsy Research
Former NINDS Director Dr. Walter Koroshetz discusses the state of epilepsy research, federal research funding, and how government, academia, industry and nonprofits do and don't collaborate, on CURE Epilepsy's Seizing Life podcast.
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1:27:47 Meet the Expert: Shyam Patel, Kevin Strauss, Manar Zaghlula
Shyam Patel, Kevin Strauss and Manar Zaghlula discuss economic and reimbursement perspectives on gene editing therapies.
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2:27 Exclusive: Death of Girl in Chinese Gene-Editing Trial Was Never Made Public
Meagan Cantwell, a video producer at Science, reports on the death of a girl in a Chinese gene-editing trial that was never made public.
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2:16 Alyssa's Story: A Base-Edited CAR-T Cell Therapy Trial
Great Ormond Street Hospital introduces Alyssa, 13, who received base-edited cell therapy for T-cell acute lymphoblastic leukaemia in a GOSH and University College London clinical trial after other treatments failed, and the research team behind the trial.
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53:53 Cellectis Clinical Pipeline Overview
Cellectis Chief Medical Officer Carrie Brownstein and Mark Frattini, SVP of Clinical and Translational Sciences, give an overview of the company's clinical product pipeline.
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59:30 Kaftrio and the CFTR Modulator Trials in Cystic Fibrosis
Dr Ian Balfour-Lynn, consultant in paediatric respiratory medicine at the Royal Brompton Hospital, reviews the clinical trials that led to licensing of CFTR modulators such as Kaftrio and real-world experience with them, for the Genomics Education Programme.
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59:57 Research Overview: Genetic Therapies in Cystic Fibrosis
Kara Foshay, PhD, and Katie Howe, MS, CCRC, of the Cystic Fibrosis Foundation explain RNA, gene editing and gene replacement therapies for CF, how they are developed and what their clinical trials look like.
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3:21 BRILLIANCE: CRISPR Gene Editing Trial for Inherited Blindness
Mass Eye and Ear reports results from the BRILLIANCE trial of an experimental CRISPR-based gene editing medicine for a form of Leber congenital amaurosis, which was safe and led to measurable improvements in 11 participants.
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56:46 Gene Editing and Rare Disease: ASGCT Congressional Briefing
ASGCT members, including Fyodor Urnov of UC Berkeley, Janice Chen of Mammoth Biosciences and Amy Raymond of Worldwide Clinical Trials, brief Congressional staff on gene editing, the clinical trials process and rare disease.
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1:42:41 CELLECTIS R&D Day 2025 - Replay
Cellectis presents the full Phase 1 data set for lasme-cel in B-ALL and the Phase 2 trial design, at its 2025 R&D Day.
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4:42 Gene Editing Saves Baby with Rare Genetic Disease
9NEWS on a baby with a rare genetic disease who is thriving after an experimental gene editing treatment made specifically for him, one of the first such cases described in a new study.
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5:44 Cleveland Clinic Doctors Say Gene-Editing Therapy Could Revolutionize Cholesterol Treatment
WKYC Channel 3 on a Phase 1 Cleveland Clinic trial testing whether gene editing could change how high cholesterol is managed.
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3:43 AHA 25: CRISPR-Cas9 Gene Editing Therapy Targeting ANGPTL3
Prof Stephen Nicholls of the Monash Victorian Heart Institute shares results from a first-in-human Phase 1 trial of a one-time CRISPR-Cas9 gene editing infusion targeting ANGPTL3 in patients with lipid disorders.
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59:43 Cellectis on Gene Editing
Cellectis on using synthetic biology with cell and gene therapy against cancer, across seven trials — three wholly owned and four partnered.