Non-Viral Vectors for Cystic Fibrosis Therapy: Recent Advances

2018 ◽  
Vol 07 (03) ◽  
Author(s):  
Faisal Qaisar ◽  
Anum Habib ◽  
Noor Muhammad ◽  
Zia ur Rehman
Prescriber ◽  
2016 ◽  
Vol 27 (11) ◽  
pp. 19-24
Author(s):  
Danny Buckland

2018 ◽  
Vol 29 ◽  
pp. 1-7 ◽  
Author(s):  
Michael D .Baron ◽  
Munir Iqbal ◽  
Venugopal Nair

2020 ◽  
Vol 15 (3) ◽  
pp. 174-186
Author(s):  
Antonella Tosco ◽  
Valeria R. Villella ◽  
Valeria Raia ◽  
Guido Kroemer ◽  
Luigi Maiuri

Since the identification of Cystic Fibrosis (CF) as a disease in 1938 until 2012, only therapies to treat symptoms rather than etiological therapies have been used to treat the disease. Over the last few years, new technologies have been developed, and gene editing strategies are now moving toward a one-time cure. This review will summarize recent advances in etiological therapies that target the basic defect in the CF Transmembrane Receptor (CFTR), the protein that is mutated in CF. We will discuss how newly identified compounds can directly target mutated CFTR to improve its function. Moreover, we will discuss how proteostasis regulators can modify the environment in which the mutant CFTR protein is synthesized and decayed, thus restoring CFTR function. The future of CF therapies lies in combinatory therapies that may be personalized for each CF patient.


1995 ◽  
Vol 20 (6) ◽  
pp. 304???308 ◽  
Author(s):  
Kenneth R. White ◽  
Cindy L. Munro ◽  
Rita H. Pickler

mSphere ◽  
2017 ◽  
Vol 2 (6) ◽  
Author(s):  
Quazim A. Alayo ◽  
Nicholas M. Provine ◽  
Pablo Penaloza-MacMaster

ABSTRACT The unprecedented challenges of developing effective vaccines against intracellular pathogens such as HIV, malaria, and tuberculosis have resulted in more rational approaches to vaccine development. Apart from the recent advances in the design and selection of improved epitopes and adjuvants, there are also ongoing efforts to optimize delivery platforms. The unprecedented challenges of developing effective vaccines against intracellular pathogens such as HIV, malaria, and tuberculosis have resulted in more rational approaches to vaccine development. Apart from the recent advances in the design and selection of improved epitopes and adjuvants, there are also ongoing efforts to optimize delivery platforms. Viral vectors are the best-characterized delivery tools because of their intrinsic adjuvant capability, unique cellular tropism, and ability to trigger robust adaptive immune responses. However, a known limitation of viral vectors is preexisting immunity, and ongoing efforts are aimed at developing novel vector platforms with lower seroprevalence. It is also becoming increasingly clear that different vectors, even those derived from phylogenetically similar viruses, can elicit substantially distinct immune responses, in terms of quantity, quality, and location, which can ultimately affect immune protection. This review provides a summary of the status of viral vector development for HIV vaccines, with a particular focus on novel viral vectors and the types of adaptive immune responses that they induce.


Neuron ◽  
2020 ◽  
Vol 107 (6) ◽  
pp. 1029-1047 ◽  
Author(s):  
Xiangmin Xu ◽  
Todd C. Holmes ◽  
Min-Hua Luo ◽  
Kevin T. Beier ◽  
Gregory D. Horwitz ◽  
...  

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