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UPenn's Kiran Musunuru, a human geneticist and practicing cardiologist who has pioneered the translation of gene- and base-editing approaches, talks to JC and Andy about the latest clinical results and modalities discussed at the 2024 Keystone symposium on Precision Genome Engineering.

4:07 Impacting patients
6:44 In vivo editing in different liver diseases
11:37 The FDA stance on programmable therapy
19:41 Base-editors march into the clinic
25:54 Multiplexing with base editors
28:57 Reaching broader patient populations
33:32 Investigator-initiated trials
39:27 Prime and epigenetic editing
44:34 Excitement around Bridge RNAs

47:15 Kiran’s mocktail

Bloody Mary 3 Ways
4oz (120 ml) tomato juice
1/2oz (15 ml) fresh lemon juice
1/4oz (7 ml) Worcestershire sauce
1/2 barspoon (3 ml) prepared horseradish, or to taste
2 dashes Tabasco, or to taste
Celery stick, for garnish
Salt and freshly ground pepper
Your choice of pickled vegetables, skewered on a cocktail pick, for garnish

DIRECTIONS: Add the tomato juice, lemon juice, Worcestershire, horseradish, and Tabasco to a shaker tin with ice and gently shake for 5 seconds. Strain into a chilled double rocks glass over a large ice cube. Garnish with the celery stick, salt, pepper, and pickled vegetables and serve.

For alcoholophiles, add 2oz (60 ml) vodka to the tomato juice, lemon, Worcestershire, horseradish and Tabasco. Enjoy!

Refs:
Gilmore et al. CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis. N Engl J Med 385, 493-502 (2021) DOI: 10.1056/NEJMoa2107454

Chiesa et al . Base-edited CAR7 T cells for relapsed T-cell acute lymphoblastic leukemia. N Engl J Med 389, 899-910 (2023) DOI: 10.1056/NEJMoa2300709

Longhurst et al. CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema N Engl J Med 390, 432-441 (2024) DOI: 10.1056/NEJMoa2309149

Durrant et al. Bridge RNAs direct modular and programmable recombination of target and donor DNA. https://www.biorxiv.org/content/10.1101/2024.01.24.577089v1

Keystone Meeting on Precision Genome Engineering

Somatic Cell Genome Editing Consortium

The Mixer music “Pour Me Another” courtesy of Smooth Moves!