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Jason Comander, MD, PhD, performs the procedure to deliver the CRISPR-based medicine as part of the BRILLIANCE trial in September 2020 at Mass Eye and Ear. Credit: Mass Eye and Ear.

All 14 trial participants, including 12 adults (ages 17 to 63) and two children (ages 10 and 14), were born with a form of Leber Congenital Amaurosis (LCA) caused by mutations in the centrosomal protein 290 (CEP290) gene. They underwent a single injection of a CRISPR/Cas9 genome editing medicine, EDIT-101 in one eye via a specialized surgical procedure. This trial, which included the first patient to ever receive a CRISPR-based investigational medicine directly inside the body, focused primarily on safety with a secondary analysis for efficacy.

No serious treatment or procedure-related adverse events were reported, nor were there any dose-limiting toxicities. For efficacy, the researchers looked at four measures: best-corrected visual acuity (BCVA); dark-adapted full-field stimulus testing (FST), visual function navigation (VNC, as measured by a maze participants completed), and vision-related quality of life.

New tech aids stroke recovery:

A groundbreaking new robotic hand that can feel touch like a human one and pave the way for new prosthetics and robots.

Summary: Researchers use AI to reveal distinct cellular-level differences in the brains of men and women, focusing on white matter. These findings show AI can accurately identify sex-based brain patterns invisible to human eyes.

The study suggests that understanding these differences can enhance diagnostic tools and treatments for brain disorders. This research emphasizes the need for diversity in brain studies to ensure comprehensive insights into neurological diseases.

A simple and robust strategy developed by KAUST scientists could help to improve the safety and accuracy of CRISPR gene editing, a tool that is already approved for clinical use for the treatment of inherited blood disorders.

Immunotherapy can boost the survival of early-stage lung cancer patients eligible for surgery when it’s combined with chemotherapy, a new clinical trial reports.

Those who got immunotherapy before and after surgery — along with pre-surgical chemo — had a 42% lower risk of cancer progression, recurrence or death than those who only received chemo, according to findings published Wednesday in the New England Journal of Medicine.

Further, about 25% of those who got immunotherapy and chemo had no cancer at all remaining after their surgery, compared with about 5% of those who got chemo alone.

Carl H. Johnson, Cornelius Vanderbilt Professor of Biological Sciences, along with a team of Vanderbilt scientists, have succeeded in adjusting the daily biological clock of cyanobacteria, making the blue-green algae a more prolific producer of renewable fuels, chemicals, and pharmaceuticals like insulin.

In episode 267 of the Stem Cell Podcast, we chat with Dr. Shankar Srinivas, a Professor of Developmental Biology in the Department of Physiology Anatomy and Genetics based in the Institute for Developmental and Regenerative Medicine at the University of Oxford. He is also a Zeitlyn Fellow and Tutor in Medicine at Jesus College. Using mouse and human embryos as model systems, his group looks at the control of patterning and morphogenesis during the establishment of the anterior-posterior axis, gastrulation, and early cardiogenesis. He discusses how tissues respond to forces during early development, characterizing cardiac progenitors, and training internationally.

Roundup Papers:
2:26 https://bit.ly/3yeD3ms.
7:14 https://bit.ly/4dKJ7nd.
19:06 https://go.nature.com/3V2SNSo.
27:10 https://go.nature.com/4dnC43H

Continue reading “The Stem Cell Podcast” »

Disk-related back pain may one day meet its therapeutic match: Gene therapy delivered by naturally derived nanocarriers that, a new study shows, repairs damaged disks in the spine and lowers pain symptoms in mice.

In plants and animals, the basic packaging units of DNA, which carry genetic information, are the so-called nucleosomes. A nucleosome consists of a segment of DNA wound around eight proteins known as histones.