Nanoblades: gene editing scissors for genome surgery
What is genome editing?
Genome editing technologies allow genetic material to be removed, added, or altered at a specific location in the genome to develop life-changing therapies for the most challenging genetic disorders.
What can out platform do for you?
Our genome editing platform provides Heidelberger scientists a wide range of tools (CRISPR/Cas systems, base editiors, prime editors, Epi-genome editors and recombinases) that enable flexible editing and unprecedented access to the genome. In addition, we offer broad and effective delivery options, including plasmids, IVT-mRNA, Ribonucleoprotein (RNP), Lentiviral and AAV systems.
CRISPR/Cas Editing:
CRISPR/Cas create double-strand breaks (DSBs) at the target site, enabling gene knock-out (KO) or knock-in (KI) using a DNA template
Base Editing:
Base editing enables the conversion of one nucleotide into another without cutting both strands of DNA. This is achieved by coupling a nickase (a Cas protein modified to cut only one DNA strand; nCas) with a deaminase that edits the target nucleotide.
ABE –> A-to-G
CBE –> C-to-T
Prime Editing:
Prime editing pairs a nickase (nCas) with a reverse transcriptase. It cuts a single DNA strand and uses pegRNA to achieve áccurate genetic changes. Re-writing of DNA, including small deletions, insertion and all 12 nucleotide substitutions.
CRISPRa & CRISPRi:
CRISPRa and CRISPRi use catalytically inactive Cas proteins (dCas) fused to epigenetic effector domains to regulate gene expression. dCas binds to gene promoters, where the effector domains either activate or repress transcription by modifying the epigenetic landscape around the target site.
- CRISPRa: Transcriptional activation – enhances gene expression
- CRISPRi: Transcriptional interference – suppresses gene expression
Recombinases:
Site-specific recombinases (SSRs) are DNA-modifying enzymes that mediate precise DNA cleavage, strand exchange, and rejoining at specific recognition sites.
Tyrosine-SSRs (T-SSRs), such as the Cre/loxP system, are effective at excising DNA but inefficient for integrating.
Serine-SSRs (S-SSRs), known as serine integrases, are highly unidirectional and can reverse only in the presence of a single accessory protein