Use of a tailored recombinases for the treatment of retroviral infections

Simple SummaryContent extracted from patent full text and abstract with AI.

This patent describes methods and compositions for creating tailored recombinases—engineered enzymes that specifically recognize and cut out proviral DNA integrated into the genome of a host cell, such as human cells infected with retroviruses like HIV. The central novelty is the use of directed evolution to generate recombinases that can recognize and recombine naturally occurring, asymmetric DNA sequences within the long terminal repeats (LTRs) of proviral DNA, enabling targeted excision of the viral genetic material. The result is an approach that can physically remove the latent, integrated viral DNA from host cells, offering a method for potentially curing retroviral infections rather than simply suppressing viral replication.

Use CasesContent extracted from patent full text and abstract with AI.

• Therapeutic removal of latent HIV DNA from the genomes of infected patients' cells, as a part of or in addition to standard antiretroviral therapy.
• Development of personalized therapies for other retroviral infections (e.g. HTLV, FIV) by tailoring recombinases to the viral sequences present in individual patients.
• Production of gene therapy vectors (ex vivo or in vivo) for excising integrated forms of harmful retroviruses from human or animal cells.
• Potential use in transforming adult stem cells ex vivo to resist or be purged of latent retroviral infections before transplantation.
• Enabling research tools for studying proviral integration and excision in cellular models.

BenefitsContent extracted from patent full text and abstract with AI.

• Targets the genetic reservoir of retroviruses (such as HIV) that conventional drugs cannot reach, offering a path toward eradication of infection rather than continuous suppression.
• Reduces or eliminates the need for lifelong antiretroviral therapy and its associated side effects and resistance issues.
• Highly specific—engineered recombinases recognize only the intended asymmetric target sequences in proviral DNA, minimizing off-target effects.
• Adaptable/personalizable—the method allows development of recombinases for different retroviral strains or patient-specific viral DNA sequences.
• Can be delivered via various gene therapy methods (viral vectors, stem cells, proteins), making it flexible for clinical translation.
• Demonstrated effectiveness in removing proviral DNA without adversely affecting host cell viability.

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Inventors & Applicants

Patent Abstract

The present invention is directed to a method for preparing an expression vector encoding a tailored recombinase, wherein said tailored recombinase recombines asymmetric target sites within the LTR of proviral DNA of a retrovirus inserted into the genome of a host cell and is useful as means for excising the provirus from the genome of the host cell. The present invention further relates to an in vitro-method of optimising the treatment of a retroviral infection of a subject and to the use of tailored recombinases for the preparation of pharmaceutical compositions for reducing the viral load in a subjected infected by a retrovirus.