Novel version of CRISPR gene-editing protein engineered
Researchers have developed a new, compact EbCas12a variant that can be packaged into an all-in-one AAV system with its crRNA.
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Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
Researchers have developed a new, compact EbCas12a variant that can be packaged into an all-in-one AAV system with its crRNA.
In this Q&A, Erik Wiklund, CEO of Circio, explains the key findings of their circVec circular RNA platform technology, why they chose AAV-based gene therapy for AATD as the lead programme, and their plans for the future to enhance the potency and reduce the cost of current gold-standard gene therapy.
The experimental therapy eliminated 90 percent of HSV-1 after facial infection and 97 percent of HSV-1 after genital infection.
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Organoid models enabled the researchers to study the effectiveness of eukaryotic ribosomal selective glycoside drugs on PKD cyst formation.
Use of the whole liver could revolutionise the development of viral vectors, providing more effective treatments for inherited diseases.
Researchers discover that fusion proteins and a gene regulatory protein complex interact through disordered domains.
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Scientists have targeted the SMRT-retinoic acid receptor interaction, which could provide a reversible and non-hormonal male contraceptive method.
Researchers have created a nanomedicine loaded with siRNAs, which demonstrated a 73 percent reduction in HIV replication.
Introducing Dr Beate Mueller-Tiemann, whose journey spans continents and disciplines, with a passion for biochemistry and molecular biology since her high school days. From elucidating pivotal proteins in cancer to monoclonal antibody discovery, Dr Mueller-Tiemann's contributions have not only transformed our understanding of diseases but have also fuelled research in…
The new study suggests that this gene therapy method may combat ARVC in both early and more advanced stages of the condition.
AcrlC8 and AcrlC9 prevent the CRISPR-Cas3 machine from binding to its DNA target site, providing a safer way to engineer the genome.
Tips and tricks for fully leveraging Advanced Flow Cytometry.