Researcher Howard Gendelman of the Center for Neurodegenerative Diseases says that the good results do not necessarily mean that it also works for people. In addition, Dr. Khalili is a co-founder, scientific advisor, member of the Board of Directors, and has received equity and monetary compensation from Excision BioTherapeutics, a biotech start-up which has licensed the viral gene editing technology from Temple University for commercial development and clinical trials. Drs. He said it was the most interesting and important therapy-related research advance he had seen in many years.
"Our study shows that treatment to suppress HIV replication and gene editing therapy, when given sequentially, can eliminate HIV from cells and organs of infected animals", said Dr. Kamel Khalili, who leads the LKSOM team.
Drs. Gendelman and Khalili were senior investigators on the new study. Their findings were published on Tuesday in Nature Communications. However, it is unclear whether this treatment will cure HIV in human beings.
The newly modified drug was packaged into nanocrystals, which can be delivered to the HIV infected tissues and slowly release the drug for weeks. This is much more successful as a long-term strategy than the antiretrovirals on their own, which are the most effective treatment now available for HIV patients.
To begin with, the scientists administered the LASER ART to slow down the HIV growth, then they added the CRISPR treatment as "chemical scissors" to "eliminate the residual integrated HIV DNA still present", said Gendelman.
The next step is for the company to enter into clinical trials with its HIV-1 targeted CRISPR platform, giving Excision BioTherapeutics the first effort to fully remove/excise the HIV-1 genome from all human cells and tissues.
Conventionally, ART alone is compelling with putting the HIV at bay; it is sufficient enough to keep it at low levels that they're undetectable in the blood, which drastically lowers the chance of spreading the virus during sexual activity or transfusions. CRISPR-Cas9 is found effective with fewer cells, which the LASER ART did great.
Researchers at University of Nebraska Medical Center are taking a critical step toward eliminating the HIV virus.
In previous work, Dr Khalili's team used CRISPR-Cas9 technology to develop a novel gene editing and gene therapy delivery system aimed at removing HIV DNA from genomes harboring the virus.
American scientists have succeeded in destroying HIV in cells of mice.
What we really need is a therapy that completely eliminates HIV from the body, which is precisely what Khalili and his colleagues have been working on for the past several years.
At the end of the treatment period, mice were examined.
"Those early cases were patients of mine", Gendelman said. It's unclear why specific animals were cleared of the infection and why others weren't.
Nicholas Snow is a Media Activist who has lived with HIV for more than a decade, he said these studies give him hope.