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For FSHD gene therapy you need to "Have-a-Little-Heart"

For FSHD gene therapy you need to "Have-a-Little-Heart"

Update: 2024-10-11

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"You know the Greeks didn't write obituaries. They only asked one question after a man died: 'Did he have passion?'. " Today we are joined by our CRISPR Goddess to discuss her new FSHD optimized gene therapy cassette that is stronger, safer, more compact and all around better than anything out there for use in FSHD and neuromuscular gene therapy approaches.

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In Channel

CRISPR technology for FSHD with Dr. Charis Himeda

CRISPR technology for FSHD with Dr. Charis Himeda

2022-03-0201:31:38

All-in-one version of our evaluation of EPI-321 data and trial

All-in-one version of our evaluation of EPI-321 data and trial

2025-09-1401:35:42

Our evaluation and opinions of the EPI-321 clinical trial design and potential based on the pre-clinical data

Our evaluation and opinions of the EPI-321 clinical trial design and potential based on the pre-clinical data

2025-09-0645:52

Our scientific evaluation of and opinions on the EPI-321 publicly available preclinical data

Our scientific evaluation of and opinions on the EPI-321 publicly available preclinical data

2025-09-0602:04:01

Prequel for the Epicrispr Bio data and clinical trial discussion

Prequel for the Epicrispr Bio data and clinical trial discussion

2025-09-0601:23:23

The nuts and bolts of gene therapy for FSHD (Part 2 of 2)

The nuts and bolts of gene therapy for FSHD (Part 2 of 2)

2025-08-1701:33:55

The nuts and bolts of gene therapy for FSHD (Part 1 of 2)

The nuts and bolts of gene therapy for FSHD (Part 1 of 2)

2025-08-1501:19:44

Part 2 of our discussion with our teen FSHD advocates

Part 2 of our discussion with our teen FSHD advocates

2025-07-3041:39

Teens promoting their "Cure FSHD for All" initiative, podcast part 1

Teens promoting their "Cure FSHD for All" initiative, podcast part 1

2025-07-2801:19:15

MyFSHD is worldwide on FSHD Day

MyFSHD is worldwide on FSHD Day

2025-06-2401:19:22

Our take on Avidity's FORTITUDE phase 1/2 trial data

Our take on Avidity's FORTITUDE phase 1/2 trial data

2025-06-1154:59

Options for nutrition, supplements, and exercise for FSHDers with Tamara Gottlieb (Part 2)

Options for nutrition, supplements, and exercise for FSHDers with Tamara Gottlieb (Part 2)

2025-04-2501:05:00

Options for nutrition, supplements, and exercise for FSHDers with Tamara Gottlieb (Part 1)

Options for nutrition, supplements, and exercise for FSHDers with Tamara Gottlieb (Part 1)

2025-04-1850:35

FaceToned® Exercises for the Face with Carme Farré

FaceToned® Exercises for the Face with Carme Farré

2025-04-1201:06:28

Brad, our angry Dad with your questions (pt 2)

Brad, our angry Dad with your questions (pt 2)

2025-04-0301:04:33

Brad, our angry Dad is back with your questions (Pt 1)

Brad, our angry Dad is back with your questions (Pt 1)

2025-03-2501:09:13

The science and the foundational funding leading to the current technologies in FSHD clinical trials.

The science and the foundational funding leading to the current technologies in FSHD clinical trials.

2025-03-1501:49:10

Friends and funding.

Friends and funding.

2025-02-2301:34:06

For FSHD gene therapy you need to "Have-a-Little-Heart"

For FSHD gene therapy you need to "Have-a-Little-Heart"

2024-10-1132:02

Fulcrum follow-up with our Angry Dad

Fulcrum follow-up with our Angry Dad

2024-09-2801:37:03

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For FSHD gene therapy you need to "Have-a-Little-Heart"

For FSHD gene therapy you need to "Have-a-Little-Heart"

Peter L Jones, PhD