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BioWorld - Tuesday, July 8, 2025
Home » Topics » Disease categories and therapies » Genetic/congenital

Genetic/congenital
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Aldh1a2 in rabbits vs. mice

Turn on vitamin A pathway and regenerate an ear

July 8, 2025
By Mar de Miguel
No Comments
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.
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Gene editing illustration
Aging

Specific inhibition of progerin production through CRISPR-Cas13 editing to treat Hutchinson-Gilford progeria syndrome

July 8, 2025
No Comments
Hutchinson-Gilford progeria syndrome (HGPS), an extremely rare genetic disorder, arises when a silent point mutation in the gene encoding the nuclear envelope protein lamin A, LMNA, leads to abnormal splicing of LMNA mRNA.
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Illustration of heart analysis for DNA and drug impacts
Cardiovascular

From maps to gene therapies, who’s who in cardiovascular disease

July 8, 2025
By Mar de Miguel
No Comments
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.
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Aldh1a2 in rabbits vs. mice

Turn on vitamin A pathway and regenerate an ear

July 2, 2025
By Mar de Miguel
No Comments
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.
Read More
Aldh1a2 in rabbits vs. mice
Genetic/congenital

Turn on vitamin A pathway and regenerate an ear

July 1, 2025
By Mar de Miguel
No Comments
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.
Read More
Ocular

AAV8-RK-hBBS10 gene therapy restores vision in Bardet-Biedl syndrome type 10 model

May 29, 2025
No Comments
Bardet-Biedl syndrome (BBS) is a group of rare autosomal recessive ciliopathies characterized by dysfunction of primary cilia, which affects multiple organ systems and leads to early-onset obesity, progressive retinal degeneration resulting in vision loss or blindness, and renal abnormalities that may progress to renal failure. Mutations in the BBS10 gene are the second most prevalent cause of BBS, accounting for over 20% of cases.
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Heart scientific overlay
Cardiovascular

Frontera’s FT-017 exerts robust activity in hypertrophic cardiomyopathy

May 27, 2025
No Comments
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).
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DNA data illustration
Genetic/congenital

Latus Bio’s LTS-101 is a potential therapeutic approach for Batten disease

May 26, 2025
No Comments
Latus Bio Inc. is developing a new gene therapy, LTS-101, for the treatment of neuronal ceroid lipofuscinosis type2 (CLN2), a form of Batten disease characterized by deficiency in the tripeptidyl peptidase 1 (TPP1) protein that leads to lysosomal dysfunction and neurodegeneration.
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Sickle cell illustration
Cancer

Study probes link between sickle cell disease and kidney cancer

May 26, 2025
By Anette Breindl
No Comments
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.
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Neurology/psychiatric

Latus Bio presents novel therapy for Huntington’s disease

May 23, 2025
No Comments
Researchers from Latus Bio Inc. have developed a novel adeno-associated virus (AAV) therapy for treating Huntington’s disease.
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