gene and cell therapy
Recently Published Documents


TOTAL DOCUMENTS

221
(FIVE YEARS 48)

H-INDEX

18
(FIVE YEARS 2)

2022 ◽  
pp. 381-420
Author(s):  
Sarfaraz K. Niazi

2021 ◽  
Vol 67 (6) ◽  
pp. 80-89
Author(s):  
O. V. Glazova ◽  
M. V. Vorontsova ◽  
N. Sakr ◽  
L. V. Shevkova ◽  
N. A. Onyanov ◽  
...  

Our current understanding of the molecular and cellular mechanisms in tissues and organs during normal and pathological conditions opens up substantial prospects for the development of novel approaches to treatment of various diseases. For instance, lifelong replacement therapy is no longer mandatory for the management of some monogenic hereditary diseases. Genome editing techniques that have emerged in the last decade are being actively investigated as tools for correcting mutations in affected organs. Furthermore, new protocols for obtaining various types of human and animal cells and cellular systems are evolving, increasingly reflecting the real structures in vivo. These methods, together with the accompanying gene and cell therapy, are being actively developed and several approaches are already undergoing clinical trials. Adrenal insufficiency caused by a variety of factors can potentially be the target of such therapeutic strategies. The adrenal gland is a highly organized organ, with multiple structural components interacting with each other via a complex network of endocrine and paracrine signals. This review summarizes the findings of studies in the field of structural organization and functioning of the adrenal gland at the molecular level, as well as the modern approaches to the treatment of adrenal pathologies.


2021 ◽  
Author(s):  
Sarfaraz Niazi ◽  
Sunitha Lokesh

2021 ◽  
Vol 32 (23-24) ◽  
pp. 1425-1426
Author(s):  
Gloria González Aseguinolaza ◽  
Ander Izeta ◽  
Francisco Martin Molina

Author(s):  
Uta Griesenbach ◽  
Rafael J. Yáñez-Muñoz

2021 ◽  
Vol 32 (19-20) ◽  
pp. 986-986
Author(s):  
Annakaisa Tirronen ◽  
Seppo Ylä-Herttuala

2021 ◽  
Vol 32 (19-20) ◽  
pp. 979-982
Author(s):  
Hildegard Büning ◽  
Elizabeth Wilson ◽  
Juan Bueren ◽  
Axel Schambach ◽  
Alberto Auricchio

2021 ◽  
Author(s):  
Alexis Cockroft ◽  
Alison Wilson

Publicly available summaries from Marketing Authorization Applications for gene and cell therapy products (advanced therapies) were evaluated to explore data expectations for product characteristics pre and post changes (comparability). Public assessment reports were used to analyze trends in information requests from regulators concerning comparability from current commercial advanced therapies. In the analysis, 12 products approved in the USA and EU were included. Inadequacies were highlighted for comparability data (six products); additional information requests (five products) and major objections were identified relating to comparability (two products, EU). Postapproval authorization obligations were imposed for six products. Comparability data are essential component for regulatory applications and public assessment reports provide a valuable source of insight into regulators’ expectations.


Sign in / Sign up

Export Citation Format

Share Document