New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.

New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.

New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria. The advance opens the door to the development of programmable methods for rearranging DNA, using recombinase enzymes guided by RNA. The two different approaches to using insertion sequences (IS) – some of the simplest and most compact mobile genetic elements – are described in two papers published in Nature and Nature Communications.

Genome editing specialist Tome Biosciences Inc. now has all the bases covered, after arriving on the scene in December with $213 million funding and three weeks later announcing the acquisition of fellow precision editing company, Replace Therapeutics Inc. for up to $185 million.

Research led by St. Jude Children’s Research Hospital and Harvard University shows base-editing approaches could be more effective than CRISPR-Cas9 gene-editing approaches for treating conditions such as sickle cell disease and β-thalassemia. Writing in the July 3, 2023, issue of Nature Genetics, the researchers compared three base-editing approaches with two CRISPR-Cas9 approaches to increasing levels of fetal hemoglobin in CD34+ hematopoietic stem and progenitor cells, and found one of the base-editing approaches was the most potent.

Vesigen Therapeutics Inc. has been awarded a grant by the Friedreich’s Ataxia Research Alliance (FARA) to develop a targeted genome editing therapeutic strategy for Friedreich’s ataxia.

The researcher who pioneered prenatal surgery to correct neural tube defects has turned her attention to using CRISPR-edited gene therapies to correct severe monogenic diseases in utero. The availability of prenatal genetic diagnosis and advances in treating fetuses, and also in gene therapy/gene editing, make it possible to repair almost any defect in the genetic code. At the same time, there is a clear rationale for intervening before birth, Tippi MacKenzie, professor of surgery at UCSF’s School of Medicine, told attendees of the third International Human Genome Editing Conference in London on March 7.

Programmable genome insertion of long DNA sequences, useful for both gene therapy and basic research, commonly relies on cellular responses to double-strand breaks (DSBs) using programmable nucleases, such as CRISPR-Cas9, for induction of repair pathways such as non-homologous end joining (NHEJ). To overcome the current limitations of gene integration approaches, scientists from the Massachusetts Institute of Technology and colleagues developed a new strategy based on advances in programmable CRISPR-based gene editing, such as prime editing, together with the application of precise site-specific integrases.

Increased payload capacity for gene therapies, off-the-shelf genome-engineered allogeneic cell therapies, reduced cost of goods and faster bioprocesses, are promised by “big DNA” specialist Replay Holdings LLC. The newco arrived with a $55 million seed round and having assembled a portfolio of technologies for writing and delivering large pieces of DNA.

Brain-wide genome editing via a single systemic dose of modified adeno-associated virus variants that cross the blood-brain barrier may represent a promising new approach for the development of disease-modifying treatments for familial Alzheimer's disease.