Results 61 to 70 of about 7,093,593 (187)

What Makes an “Ideal” Cell Line for Recombinant Adeno‐Associated Virus Production?

Biotechnology and Bioengineering, EarlyView.
Several host cell types have been used to produce rAAVs to date. Cell line‐specific traits that are beneficial in the context of rAAV manufacturing are reviewed here, with the goal of developing a consensus on the ‘ideal’ characteristics that an rAAV production host should possess (created with Biorender.com).
James Conheady, Nicholas Donohue, Alexandra Bogdanovic, Sharon Davin, Brian Glennon, Niall Barron, C. Mark Smales   +6 more
wiley   +1 more source

Cell and Gene Therapies Manufacturing Challenges and Integrated Good Manufacturing Practices Solutions: A Lifecycle Perspective

Biotechnology and Bioengineering, EarlyView.
Lifecycle perspective on cell and gene therapy manufacturing challenges and enabling GMP solutions. ABSTRACT Cell and gene therapies (CGTs) are revolutionizing modern medicine; however, making these advanced medicines scalable and readily available to commercial manufacturers worldwide is a major challenge. The number of approved CGT products continues
Rajath Samaga, Udisha Singh, Sachin Dattram Pawar   +2 more
wiley   +1 more source

Influence of Hypothermic Storage Fluids on Mesenchymal Stem Cell Stability: A Comprehensive Review and Personal Experience

Cells, 2021
Mesenchymal stem cells have generated a great deal of interest due to their potential use in regenerative medicine and tissue engineering. Examples illustrating their therapeutic value across various in vivo models are demonstrated in the literature ...
Aneta Ścieżyńska, Marta Soszyńska, Patrycja Szpak, Natalia Krześniak, Jacek Malejczyk, Ilona Kalaszczyńska   +5 more
doaj   +1 more source

Decellularised Corneal Stromal Lamina With or Without Autologous Adipose‐Derived Adult Stem Cells for Advanced Keratoconus: 1‐Year Results of a Phase I/IIa Clinical Trial

Clinical &Experimental Ophthalmology, EarlyView.
ABSTRACT Background To report the 1‐year clinical outcomes of decellularised human corneal stromal lamina implantation, with or without autologous adipose‐derived adult stem cells (ADASCs), for advanced keratoconus in a European cohort. Methods In this prospective, randomised Phase I/IIa trial, 10 eyes of 10 patients were allocated to decellularised ...
Hao Zhang, Jorge L. Alió del Barrio, Alejandra E. Rodriguez, Rafael I. Barraquer, Francisco Arnalich‐Montiel, David Mingo‐Botín, María Eugenia Fernández Santos, Jorge L. Alió   +7 more
wiley   +1 more source

Preclinical study for treatment of hypospadias by advanced therapy medicinal products [PDF]

, 2020
Purpose This paper explores the feasibility of a new therapy for the treatment of hypospadias patients. Hypospadias is a very common congenital malformation of male genitals, with very high rate of recurrences after surgery.
Bianchi E., Rizzo M., Lazzeri, M., Trombetta, C., Fantacci M., Galaverni G., Losi, L., Lazzeri M., Attico, E., Trombetta C., Balò, S., Pellegrini, G., Ribbene, A., Corradini F., Manfredini, R., Manfredini R., Pellegrini G., Barbagli, G., Losi L., Galaverni, G., Melonari, M., Sceberras V., Ribbene A., Bianchi, E., Fantacci, M., Barbagli G., Corradini, F., Rizzo, M., Melonari M., Sceberras, V., Attico E., Balo S.   +31 more
core   +1 more source

Bringing Gene Therapy Into Real World Clinical Practice

Haemophilia, EarlyView.
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach, Pratima Chowdary, Margareth C. Ozelo   +2 more
wiley   +1 more source

Italian Patients Journey for Gene Therapy in Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Gene therapy (GT) provides sustained FVIII levels without repeated infusions in Hemophilia A (HA) patients thus overcoming a major limitation of replacement therapy. However, issues remain e.g., patient selection criteria, duration and variability of transgene expression, quality of life and long‐term safety.
Giovanni Di Minno, Gaia Spadarella, Alessandra Borchiellini, Anna Borrelli, Andrea Buzzi, Giancarlo Castaman, Michele Cecchi, Raimondo De Cristofaro, Vincenzo La Mura, Angelo Lupi, Giuseppe Malcangi, Maria Elisa Mancuso, Maria Francesca Mansueto, Cristina Santoro, Rita Carlotta Santoro, Luigi Piero Solimeno, Annarita Tagliaferri, Ezio Zanon, Isabella Garagiola, Flora Peyvandi   +19 more
wiley   +1 more source

Development of a proficiency testing platform for advanced therapies medicinal products (ATMPs)

Cytotherapy
Background: The development of Advanced Therapy Medicinal Products (ATMPs) is driving major advances in regenerative medicine, particularly in cellular immunotherapies. However, the reliable application of quality control methods remains a major challenge due to the inherent complexity of biotechnological products, especially when assays involve living
Jesús Chaparro-García, Rafael Maldonado-Sánchez, Anaïck Moisan, Marga Blanco, Cristina Antúnez, Jorge Guerrero, Natividad Fernández, Marcela Eisman, Elena Tristante, Martínez-Lorenzo María José, Elena Vuelta, Javier Calvo, Purificación Catalina, Alicia Moreno Pantoja, Mónica Santos González, María Bermejo, Blanca Arribas-Arribas, Laura Leyva, Antonio Rodríguez-Acosta   +18 more
openaire   +2 more sources

Harnessing the Therapeutic Potential of Cell Secretomes and Extracellular Vesicles for Craniofacial Regenerative Applications

Journal of Periodontal Research, EarlyView.
The scoping review summarizes the current preclinical and clinical evidence for the use of “cell‐free” therapies in craniofacial (periodontal, bone and soft‐tissue) regeneration. It also aims to highlight key challenges and strategies towards the clinical translation of these therapies.
Siddharth Shanbhag, Magdalena Mayol, Danijel Domic, Madhusudhan Reddy Bobbili, Johannes Grillari, Mariano Sanz, Reinhard Gruber   +6 more
wiley   +1 more source

Cell Therapy for Periodontal, Soft‐Tissue, and Craniofacial Regeneration

Journal of Periodontal Research, EarlyView.
The clinical translation of mesenchymal stem/stromal cells (MSCs) represents a major advancement in dentistry, especially for periodontal, soft‐tissue, and craniofacial regeneration. However, several challenges remain to be addressed, including the absence of standardised protocols, limited scalability, regulatory hurdles, a lack of well‐controlled ...
Kamal Mustafa, Shuntaro Yamada, Nerea Sanchez, Magdalena Mayol, Cecilie Gjerde, Mariano Sanz   +5 more
wiley   +1 more source