Genetic therapies in cystic fibrosis
JL Taylor-Cousar, AC Boyd, EWFW Alton… - Current Opinion in …, 2023 - journals.lww.com
Genetic therapies in cystic fibrosis : Current Opinion in Pulmonary Medicine Genetic therapies
in cystic fibrosis : Current Opinion in Pulmonary Medicine Log in or Register Subscribe to …
in cystic fibrosis : Current Opinion in Pulmonary Medicine Log in or Register Subscribe to …
A new era of targeting cystic fibrosis with non-viral delivery of genomic medicines
Cystic fibrosis (CF) is a complex genetic respiratory disorder that necessitates innovative
gene delivery strategies to address the mutations in the gene. This review delves into the …
gene delivery strategies to address the mutations in the gene. This review delves into the …
[HTML][HTML] Targeting lung cancer with clinically relevant EGFR mutations using anti-EGFR RNA aptamer
BJ Thomas, C Guldenpfennig, Y Guan… - … Therapy-Nucleic Acids, 2023 - cell.com
A significant fraction of non-small cell lung cancer (NSCLC) cases are due to oncogenic
mutations in the tyrosine kinase domain of the epidermal growth factor receptor (EGFR). Anti …
mutations in the tyrosine kinase domain of the epidermal growth factor receptor (EGFR). Anti …
Antisense oligonucleotides and their technical suitability to nebulization
LL Seidl, R Moog, KA Graeser - International Journal of Pharmaceutics, 2024 - Elsevier
In vivo studies investigating the inhalative efficacy of biotherapeutics, such as nucleic acids,
usually do not perform an aerosolization step, rather the solution is directly administered into …
usually do not perform an aerosolization step, rather the solution is directly administered into …
The safety and toxicity profile of SPL84, an inhaled antisense oligonucleotide for treatment of cystic fibrosis patients with the 3849+ 10kb C-> T mutation, supports a …
L Friedman, OB Avitzur, EO Galai, N Ferrari… - Expert Opinion on …, 2023 - Taylor & Francis
Introduction SPL84 is an inhaled antisense oligonucleotide (ASO) in development for the
treatment of cystic fibrosis (CF) patients carrying the 3849+ 10kb C-> T (3849) mutation. To …
treatment of cystic fibrosis (CF) patients carrying the 3849+ 10kb C-> T (3849) mutation. To …
Zukunft der Mukoviszidoseforschung und-therapie
SY Graeber, MA Mall - Monatsschrift Kinderheilkunde, 2024 - Springer
Mukoviszidose (zystische Fibrose, CF) ist die häufigste monogenetische Erkrankung in der
weißen Bevölkerung und wird durch Mutationen im CFTR-Gen verursacht. Die häufigste …
weißen Bevölkerung und wird durch Mutationen im CFTR-Gen verursacht. Die häufigste …