spacer
home > ebr > autumn 2017 > size matters
PUBLICATIONS
European Biopharmaceutical Review

Size Matters

Progressive research in gene and cell therapy, as well as DNA vaccination, requires safe pharmaceutical vectors. The former, in particular, is on the rise, with a huge potential for treating inherited disorders. However, the advances in this area depend on improved vector designs, which enable the safe delivery of therapeutics into target cells (1). The novel transfer vectors should be direct, tissue-specific, stable and highly expressive. They have to be minimally toxic, regarding anti-vector immune responses, and should not be integrated into the host genome (2). Without integration, the toxicity is low, but this also means that long-term expression – and thus therapeutic effect in mitotic cells – may not occur because of progressive loss of vector DNA (2).

Usually, viral and non-viral vectors based on plasmid DNA are used for the transfer of genetic information into the target cells. Plasmids encode these on the intracellular production of therapeutic virus particles (3). Clinical use of plasmid DNA for gene or cell therapy, as well as genetic vaccination, has been in an inferior position to viral methods, but has made significant progress in recent years (4,5).

Challenging Transfers

One limiting factor in transgene expression is the transfer process into the cytoplasm and nucleus, as the size of the DNA is crucial (6,7). The smaller it is, the better the nuclear entry. The differing proportions between two otherwise identical plasmids, such as monomer and dimer, show the influence of this procedure (8).

Read full article from PDF >>

Rate this article You must be a member of the site to make a vote.  
Average rating:
0
     

There are no comments in regards to this article.

spacer
Tatjana Buchholz is a certified Medical Technical Laboratory Assistant. She also has a Bachelor of Science in molecular biotechnology and a Master of Science in genome-based systems biology from the Bielefeld University, Germany. Tatjana’s bachelor’s thesis was occupied with signal studies of the promoter activity of the nuclear LHC translational repressor NAB1 in the microalgae chlamydomonas reinhardtii, and her master’s thesis was concerned with MC technology optimisation. Tatjana joined PlasmidFactory as Marketing Manager in 2016.

Dr Marco Schmeer studied chemistry and holds a PhD from the University of Bielefeld, Germany. After further postdoctoral training in the fields of electroporative gene and drug transfer, he joined PlasmidFactory as Product and Project Manager in 2005, when he was made responsible for client-related plasmid and MC production projects.

Dr Martin Schleef studied biology at the universities Würzburg and Bielefeld, Germany, and holds a PhD from the latter. He received postdoctoral training from the Institut Pasteur Paris, France, and joined QIAGEN GmbH in Hilden, Germany, in 1994. Martin is founder and CEO of PlasmidFactory, which has been a prosperous biotech company in Bielefeld since 2000. He is also a lecturer at Bielefeld University.

spacer
Tatjana Buchholz
spacer
spacer
spacer
Dr Marco Schmeer
spacer
spacer
spacer
Dr Martin Schleef
spacer
spacer
Print this page
Send to a friend
Privacy statement
News and Press Releases

Clinical and Regulatory Operational Excellence Forum

1. Innovative technologies are expected to take clinical development, licensing, and other regulatory processes to the next level in the future. What do you see are the biggest challenges facing companies when trying to cope with continued data growth in a fast changing environment? Pharmaceutical companies are heavily regulated and introducing change needs proof of acceptance by the authorities. The chance that authorities do not agree with that change makes the industry risk averse. Changes in a stable GxP environment are almost by default seen as a risk, however, not adapting to new technologies should also be seen as a risk! Therefore, ICH developed the ICH Q8, Q9, Q10, Q11, and Q12 guidelines, to anticipate change and implement changes much faster, with less of a regulatory burden. Nevertheless, industry is not picking up with the desired pace. Why not?
More info >>

White Papers

Characterisation of Biopharmaceutical Proteins

Reading Scientific Services Ltd (RSSL)

Over the next five years it is anticipated that there is going to be an explosion in the numbers of biosimilar products coming to market as patents expire. Consequently, in line with regulatory guidance, there will be a commensurate need to provide full characterisation of such biopharmaceuticals. The purpose of this article is to describe the array of the more common techniques used in biopharmaceutical characterisation (typically of protein or polypeptide). For full characterisation of a protein, the protein�s primary, secondary and tertiary structure as well as its physiochemical properties should be assessed.
More info >>

 
Industry Events

Pharma Packaging and Labelling East Coast 2019

20-21 February 2019, Phildaelphia, USA

Now in its 11th year, Pharma Packaging and Labeling is back to deliver key, actionable insights into very latest regulatory requirements, technological innovations, strategic developments, and how to implement them into your packaging and labeling chain with both maximum efficiency and minimal cost.
More info >>

 

 

©2000-2011 Samedan Ltd.
Add to favourites

Print this page

Send to a friend
Privacy statement