Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...

This is an important study that identifies the developmental time window during which re-expression of TCF4 mutated in Pitt-Hopkins syndrome, can rescue phenotypic features of brain function in a TCF4 ...

Human Continuum has announced the appointment of Dr Omar Khalid Sial as Chief Scientific Officer (CSO).

BAG3 DCM is a devastating genetic heart disease caused by a mutation in the BAG3 gene and resulting in loss of heart function at a young age; despite current standard of care, almost 25% of patients ...

Retrotransposons are pivotal in human genome evolution, driving structural variation and regulatory innovation while influencing health and disease.

New study reveals how cytoplasmic and nuclear functions of USP30-AS1 converge to promote tumor growth Breast cancer progression is increasingly ...

New review unravels how mitochondrial dysfunction and cellular aging converge to drive Parkinson’s disease Parkinson’s disease (PD) is a progressive ...

Krystal is leveraging a validated, redosable platform with high margins, profitability, and multiple blockbuster catalysts in CF and NK. Read why KRYS is a Buy.

PDE6A-associated RP, a rare form of inherited retinal disease (IRD), is characterized by nyctalopia, visual field defects, and significant loss of visual acuity (VA). The disease primarily affects the ...

OGM was selected as a genome-wide, unbiased method to detect large genomic rearrangements and structural variants (SVs) with sensitivity to variant allele fractions (VAFs) as low as 5% to analyze ...

Finding Hope for Frizzle (FRRS1L), a parent-led nonprofit organization working to develop a lifesaving treatment for FRRS1L patients, and Apertura Gene Therapy, a biotechnology company developing next ...