METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION

Smart overview of the Invention

A method has been developed to prevent the transmission of HIV from a mother to her child. This involves administering a composition that contains a CRISPR-associated endonuclease and multiple guide RNAs (gRNAs) to the mother. Each gRNA is designed to target different sequences within the long terminal repeat (LTR) of the proviral DNA unique to HIV, allowing for precise cleavage of the viral genome. The process aims to excise the entire HIV-1 proviral genome, effectively eradicating it from host cells and preventing its transmission during childbirth.

Significance of CRISPR Technology

The use of CRISPR technology in this method leverages its ability to specifically target and edit genetic material. By employing a combination of gRNAs that are complementary to distinct sequences in the HIV genome, the method enhances the likelihood of successful viral genome elimination. This approach addresses a critical issue with current therapies, which often fail to eliminate latent reservoirs of the virus that can lead to ongoing infection and transmission.

Clinical Relevance

HIV/AIDS continues to be a significant global health crisis, with millions affected worldwide. Traditional treatments, such as highly active antiretroviral therapy (HAART), manage viral load but do not eradicate latent infections or prevent transmission effectively. The proposed method offers a potential breakthrough by targeting integrated proviral DNA directly, which could lead to more effective treatment strategies and possibly a cure for infected individuals.

Composition Details

The compositions described include nucleic acids encoding a CRISPR-associated endonuclease like Cas9, along with guide RNAs that target HIV sequences. These components can be formulated into pharmaceutical preparations for therapeutic use. The compositions aim to provide both prophylactic and therapeutic options for individuals at risk of or currently infected with HIV, potentially enhancing existing treatment regimens.

Research Background

This innovation has been developed with support from government research funding, highlighting its scientific foundation and potential for real-world application. The research underscores the importance of advancing methods that can effectively combat persistent viral infections like HIV, which have long posed challenges in public health due to their complex nature and ability to evade conventional therapies.