Cellular atlases and omics studies, such as genomics, transcriptomics and proteomics, have become key tools for identifying the diversity of all the elements that make up the cardiovascular system. These approaches help scientists understand how cells, genes and molecules function and interact in both healthy and diseased conditions, revealing critical points where targeted interventions could not only relieve symptoms but potentially reverse the underlying pathology at its origin.

Lizards, zebrafish, salamanders and tritons can regrow a tail, a fin, or even an entire limb after amputation. Cut a planarian into pieces, and you will end up with a bunch of them. Researchers at the National Institute of Biological Sciences in Beijing have discovered a genetic switch linked to vitamin A. After activating this pathway, they managed to regenerate the ear pinna of a mouse, an animal that previously lacked this ability.

Lizards, zebrafish, salamanders and tritons can regrow a tail, a fin, or even an entire limb after amputation. Cut a planarian into pieces, and you will end up with a bunch of them.

Frontera Therapeutics Inc. has developed FT-017, an adenovirus-associated vector(AAV)-based gene therapy that carries a human MYBPC3 optimized codon, for the treatment of hypertrophic cardiomyopathy (HCM).

Latus Bio Inc. is developing a new gene therapy, LTS-101, for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease characterized by deficiency in the tripeptidyl peptidase 1 (TPP1) protein that leads to lysosomal dysfunction and neurodegeneration.

Individuals with both sickle cell disease (SCD) and sickle cell trait are at higher risk than others of developing renal medullary cancer (RMC), the rarest and deadliest subtype of kidney cancer. Researchers at MD Anderson Cancer Center have identified the molecular mechanisms behind the increased risk, gaining new insights into antitumor immunity more generally and, potentially, new ways to treat RMC, and possibly other tumors as well.

SCD “has been studied for 30 years, but 95% of the effort [has been] working on the red blood cells … how red blood cells contribute to hypoxia and then reduce oxygen supply,” Chunru Lin told BioWorld.

The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare disorder affecting brain development for which a mouse model has finally been developed. Other times, small and large models exist, but an effective treatment remains elusive, as is the case with Krabbe disease, a fatal disease in children that could be prevented with the advances in gene therapy.

Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) marked a historic milestone this year by presenting the first case of treatment with base editors of a baby with a deadly metabolic disease.