Mount Sinai researchers develop novel therapy that could be effective in many cancers

New York, NY (July 23, 2021) — Mount Sinai researchers have developed a therapeutic agent that shows high effectiveness in vitro at disrupting a biological pathway that helps cancer survive, according to a paper published in Cancer Discovery , a…

CHOP Researchers Use Base Editing in Preclinical Model to Correct Lethal Lysosomal Storage Disease Before Birth

Adding to the growing body of literature demonstrating the feasibility of correcting lethal genetic diseases before birth, researchers at Children’s Hospital of Philadelphia (CHOP) have used DNA base editing in a prenatal mouse model to correct a lysosomal storage disease known as Hurler syndrome. Using an adenine base editor delivered in an adeno-associated viral vector, the researchers corrected the single base mutation responsible for the condition, which begins before birth and affects multiple organs, with the potential to cause death in childhood if untreated.

Innovative Gene Therapy ‘Reprograms’ Cells To Reverse Neurological Deficiencies

A new method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. The study was led by researchers at The Ohio State University Wexner Medical Center and The Ohio State University College of Medicine.

New adaptable nanoparticle platform enables enhanced delivery of gene therapies

Scientists have developed polypeptide-based materials that act as effective vectors for delivering gene therapies. The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo. The work, led by researchers from RCSI University of Medicine…

Controlling insulin production with a smartwatch

Many modern fitness trackers and smartwatches feature integrated LEDs. The green light emitted, whether continuous or pulsed, penetrates the skin and can be used to measure the wearer’s heart rate during physical activity or while at rest. These watches have…

The ISSCR releases updated guidelines for stem cell research and clinical translation

Skokie, IL – The International Society for Stem Cell Research (ISSCR), today released updated guidelines for stem cell research and its translation to medicine. The update reflects emerging advances including, stem cell-based embryo models, human embryo research, chimeras, organoids, and…

Cholesterol levels sustainably lowered using base editing

Base editing is a novel gene editing approach that can precisely change individual building blocks in a DNA sequence. By installing such a point mutation in a specific gene, an international research team led by the University of Zurich has…

Penn Medicine and Children’s Hospital of Philadelphia to Host Symposium on the Future of Cell and Gene Therapies

Penn Medicine and Children’s Hospital of Philadelphia (CHOP) will host a virtual event on May 6 and 7 that will bring together cell and gene therapy leaders from the two institutions and around the world to discuss the latest achievements in the field, novel strategies, and future developments and applications for chimeric antigen receptor, CAR, T cell therapy and more.

Better hearing with optical cochlear implants

Understanding spoken words, developing normal speech – cochlear implants enable people with profound hearing impairment to gain a great deal in terms of quality of life. However, background noises are problematic, they significantly compromise the comprehension of speech of people…

Audacious projects develop imaging technology to aid eye tissue regeneration

As regenerative therapies for blinding diseases move closer to clinical trials, the National Eye Institute’s functional imaging consortium, a part of the NEI Audacious Goals Initiative (AGI), is pioneering noninvasive technologies to monitor the function of the retina’s light-sensing neurons and their connections to the brain.

Carnegie Mellon/Yale PNA-based technique an essential part of the gene editing toolkit

In an article published in the April 8 issue of Nature , the National Institutes of Health’s Somatic Cell Gene Editing Consortium provided a detailed update on the progress of their nationwide effort to develop safer and more effective methods…