Authors: Liu YP, Berkhout B
RNA interference (RNAi) holds great promise as gene therapy approach against viral pathogens, including HIV-1. A specific anti-HIV-1 response can be induced via transfection of synthetic small interfering RNAs (siRNAs) or via intracellular transgene expression of short hairpin RNAs (shRNAs) or microRNAs (miRNAs). Both targeting of the viral mRNAs or the mRNAs for cellular co-factors that are required for viral replication have been shown successful in suppressing HIV-1 replication. However, like conventional mono-therapies, the use of a single anti-HIV-1 RNAi inducer results in the emergence of RNAi-escape mutants. To prevent viral escape, a combinatorial RNAi approach should be used in which multiple RNAi effectors against HIV-1 are simultaneously expressed…

Authors: Jafari M, Chen P
Applying RNA interference to silence a specific gene has opened a new and promising avenue of gene therapy. But a key bottleneck is the poor stability and inability of naked siRNA to translocate through cell membranes. Among several delivery systems, cationic peptides capable of penetrating cell membranes have drawn attention due to their structural and functional versatility, potential biocompatibility and ability to target cells. In this review, different classes of peptides employed in siRNA delivery are reviewed. In particular, a new class of siRNA delivery peptides with high transfection efficiency and low cytotoxicity is introduced.
PMID: 19860709 [PubMed - in process] (Source: Current Topics in Medicinal Chemistry)

“Donated lungs that have to be discarded because they are not good enough for transplant can now be repaired and made suitable for patients,” The Times has reported.
The study behind the news looks at an experimental gene therapy technique that has been tested on pig and human lungs. Under this technique, a gene known as IL-10 is introduced into the lung tissue cells to alter their behaviour. The research showed that in experimental conditions the gene combatted the damaging inflammation that sometimes causes problems in lung transplants.
The importance of the research is emphasised in a journal editorial, which points out that only 15% of donor lungs are currently suitable for transplantation, therefore this process may become important if it proves successful in future studies.
The…

Authors: Summer B. Thyme, Jordan Jarjour, Ryo Takeuchi, James J. Havranek, Justin Ashworth, Andrew M. Scharenberg, Barry L. Stoddard & David Baker
Enzymes use substrate-binding energy both to promote ground-state association and to stabilize the reaction transition state selectively. The monomeric homing endonuclease I-AniI cleaves with high sequence specificity in the centre of a 20-base-pair (bp) DNA target site, with the amino (N)-terminal domain of the enzyme making extensive binding interactions with the left (-) side of the target site and the similarly structured carboxy (C)-terminal domain interacting with the right (+) side. Here we show that, despite the approximate twofold symmetry of the enzyme–DNA complex, there is almost complete segregation of interactions responsi…

[ The following is an exact transcript of this podcast. ]Emphysema and cystic fibrosis patients who need new lungs are faced with a life-threatening problem: more than 80 percent of donated lungs can’t be used–they’re inflamed and barely functional. But a new approach, detailed this week in the new journal Science Translational Medicine , describes a novel gene therapy that can repair these damaged lungs–and make them available for transplant. [More] (Source: Scientific American Topic – Biotechnology)

For the first time, scientists in the McEwen Centre for Regenerative Medicine, University Health Network have successfully used gene therapy to repair injured human donor lungs, making them potentially suitable for transplantation into patients. (Source: Health News from Medical News Today)

Ultrasound and microbubble contrast agents have proved to be a valuable team in many diagnostic applications. But these technologies may also ultimately combine to give a significant boost to the nascent field of gene therapy, according to research presented (more) (Source: AuntMinnie.com Headlines)

New procedure could sharply increase the supply of functional lungs by preventing and possibly repairing organs damaged during harvesting. The process also may decrease rejection by the recipient.

A new gene therapy procedure to restore function in lungs damaged during harvesting from donors could make more of the organs available for transplanting, Canadian researchers reported Wednesday. (Source: Los Angeles Times – Science)

A PIONEERING technique could help repair lungs damaged during a patient’s death and make them suitable for transplant patients, scientists have shown. (Source: Scotsman.com News – Health)

Induced pluripotent stem cell (iPSC) technology has emerged as the most promising method for generating patient-specific human embryonic stem (ES) cells and adult stem cells (Takahashi et al., 2007, Cell 131:861-872; Wernig et al., , Nature 448:318-324; Park et al., 2008, Nature 451:141-146). So far, most studies of direct reprogramming have been done by using lentiviruses/retroviruses encoding the reprogramming factors. This represents a major limitation to therapeutic applications since viral integration in the host genome increases the risk of tumorigenicity, and low-level residual expression of reprogramming factors may alter the differentiation potential of the human iPSCs (hiPSCs). As a result, more attention has been paid to developing new techniques to manipulate the human genome, …