Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Promising HIV Therapy in Development: Molecular Scissors Cut Off The “Door Handle”

18.05.2015

Remove the “door handle” and stop the HIV virus that causes AIDS from entering the cell. Researchers at the University Medical Center Hamburg-Eppendorf (UKE) have developed molecular scissors that can be targeted to dissect the gene of a receptor needed by HIV to infect human immune cells.

The gene scissors, part of a “second generation of designer nuclease,” dissect more effectively than enzymes previously used in research. “During testing the dissection was successful in more than half of the cells,” reported Prof. Dr. Boris Fehse from the Interdisciplinary Clinic and Polyclinic for Stem Cell Transplantation at the UKE.

In the coming years this type of somatic therapy, the removal of immune cells, which after being treated and transfused back into the patient, could benefit those with HIV. However, further positive results from future testing are required.

“If the current pre-clinical studies confirm the safety of the procedure, then due to the high rate of efficiency, and the massively improved precision when compared to first generation nuclease, coupled with the relatively simple application, we see a real possibility that we can soon begin with clinical testing,” says Prof. Fehse, head of the Research Department for Cell and Gene Therapy. Stopping the virus at the point of entry is the most promising strategy of today’s AIDS research.

The Human Immunodeficiency Virus (HIV) requires molecules at the surface of the immune cells (more precisely: T-Helper Cells) as a portal of entry. “During the initial stages of infection the chemokine receptor, CCR5, plays an essential role as a co-receptor.” In short, if CCR5 is not present on the surface of the T-helper cell then the HIV virus is unable to dock and infect the cell.

“In cooperation with the Heinrich-Pette-Institute here in Hamburg, we are able to show that the genetic changes caused by our newly developed enzyme provide infection protection against reference strains of the HIV virus.”

Fewer Side Effects Thanks to Messenger RNA

Prof. Fehse and the lead author, Dr. Ulrike Mock, reported in the online edition of the specialist journal “Nucleic Acids Research,” that the enzyme scissors named “CCR-Uco” was successful in destroying the CCR5 gene is more that 50% of the T-cells, a process known as gene knockout. To place the molecular scissors in the T-cell, the research team used a synthetically created messenger RNA, and a gene ferry.

“The use of the messenger RNA is, from a practical standpoint, far less complicated than using a virus for gene transfer,” explained Prof. Fehse. The lifespan of the messenger RNA within the cell is much shorter than that of a virus, and the degrading process is completed faster. This means that there is a notable reduction of unwanted side effects as there is the risk that the scissor enzyme would continue to attack the gene after the therapeutic part of its function has been completed.

Chance Discovery: “The Berlin-Patient”

We discovered that the retroactive removal of the CCR5 receptor could actually cure those infected with HIV by pure chance. In North America and Northern Europe, approximately one percent of the population has no CCR5 on their immune cells as the result of a genetic defect. Therefore, these individuals are almost completely protected against the HIV infection. A patient known in research circles as “The Berlin Patient” is the only HIV infected person known worldwide to have been cured, thanks to this genetic defect. The patient developed leukemia and was treated using a stem cell transplant. In this case, the stem cell donor had this advantageous genetic defect, which was transferred to the patient through bone marrow stem cells.

References:
U. Mock et. al. mRNA transfection of a novel TAL effector nuclease (TALEN) facilitates efficient knockout of HIV co-receptor CCR51, Nucleic Acids Research, 2015 1 (DOI: 10.1093/nar/gkv469)

Contact:
Prof. Dr. Boris Fehse
Head of the Research Department for Cell and Gene Therapy
Interdisciplinary Clinic and Polyclinic for Stem Cell Transplantation
Universitätsklinikum Hamburg-Eppendorf
Martinistr. 52
20246 Hamburg
Telefon: (040) 7410-55518
E-Mail: fehse@uke.de

Saskia Lemm | idw - Informationsdienst Wissenschaft

More articles from Interdisciplinary Research:

nachricht Stanford researchers create a wireless, battery-free, biodegradable blood flow sensor
09.01.2019 | Stanford University

nachricht Description of rotating molecules made easy
21.12.2018 | Institute of Science and Technology Austria

All articles from Interdisciplinary Research >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Ten-year anniversary of the Neumayer Station III

The scientific and political community alike stress the importance of German Antarctic research

Joint Press Release from the BMBF and AWI

The Antarctic is a frigid continent south of the Antarctic Circle, where researchers are the only inhabitants. Despite the hostile conditions, here the Alfred...

Im Focus: Ultra ultrasound to transform new tech

World first experiments on sensor that may revolutionise everything from medical devices to unmanned vehicles

The new sensor - capable of detecting vibrations of living cells - may revolutionise everything from medical devices to unmanned vehicles.

Im Focus: Flying Optical Cats for Quantum Communication

Dead and alive at the same time? Researchers at the Max Planck Institute of Quantum Optics have implemented Erwin Schrödinger’s paradoxical gedanken experiment employing an entangled atom-light state.

In 1935 Erwin Schrödinger formulated a thought experiment designed to capture the paradoxical nature of quantum physics. The crucial element of this gedanken...

Im Focus: Nanocellulose for novel implants: Ears from the 3D-printer

Cellulose obtained from wood has amazing material properties. Empa researchers are now equipping the biodegradable material with additional functionalities to produce implants for cartilage diseases using 3D printing.

It all starts with an ear. Empa researcher Michael Hausmann removes the object shaped like a human ear from the 3D printer and explains:

Im Focus: Elucidating the Atomic Mechanism of Superlubricity

The phenomenon of so-called superlubricity is known, but so far the explanation at the atomic level has been missing: for example, how does extremely low friction occur in bearings? Researchers from the Fraunhofer Institutes IWM and IWS jointly deciphered a universal mechanism of superlubricity for certain diamond-like carbon layers in combination with organic lubricants. Based on this knowledge, it is now possible to formulate design rules for supra lubricating layer-lubricant combinations. The results are presented in an article in Nature Communications, volume 10.

One of the most important prerequisites for sustainable and environmentally friendly mobility is minimizing friction. Research and industry have been dedicated...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

VideoLinks
Industry & Economy
Event News

Our digital society in 2040

16.01.2019 | Event News

11th International Symposium: “Advanced Battery Power – Kraftwerk Batterie” Aachen, 3-4 April 2019

14.01.2019 | Event News

ICTM Conference 2019: Digitization emerges as an engineering trend for turbomachinery construction

12.12.2018 | Event News

 
Latest News

Additive manufacturing reflects fundamental metallurgical principles to create materials

18.01.2019 | Materials Sciences

How molecules teeter in a laser field

18.01.2019 | Life Sciences

The cytoskeleton of neurons has been found to be involved in Alzheimer's disease

18.01.2019 | Health and Medicine

VideoLinks
Science & Research
Overview of more VideoLinks >>>