“Intelligence et humanité sont les deux impressions qui me sont restées de mes échanges avec Raphaël alors qu’il était mon étudiant. Deux impressions qui se concrétisaient alors par par des questions et des propos habituellement posées /tenus par des séniors qui aspirent à leur meilleure version, et avec certitude par une personne mue par des motivations humaines. Ainsi je ne suis pas surprise mais ravie de voir la suite de son parcours avec JobTaste. Je lui souhaite le meilleur et le recommande vivement à qui est en quête de sens et d’une meilleure société.”
Raphael Schatz 🧡
Strasbourg et périphérie
3 k abonnés
+ de 500 relations
À propos
Depuis longtemps, je cherche mon épanouissement professionnel.
Je me suis rendu…
Services
Expérience
Formation
Licences et certifications
Publications
-
The challenge of gene therapy for neurological diseases: strategies and tools to achieve efficient delivery to the central nervous system
Human Gene Therapy
For more than ten years, gene therapy for neurological diseases has experienced intensive research growth and more recently therapeutic interventions for multiple indications. Beneficial results in several phase 1/2 clinical studies, together with improved vector technology have advanced gene therapy for the central nervous system (CNS) in a new era of development. While most initial strategies have focused on orphan genetic diseases, such as lysosomal storage diseases, more complex and…
For more than ten years, gene therapy for neurological diseases has experienced intensive research growth and more recently therapeutic interventions for multiple indications. Beneficial results in several phase 1/2 clinical studies, together with improved vector technology have advanced gene therapy for the central nervous system (CNS) in a new era of development. While most initial strategies have focused on orphan genetic diseases, such as lysosomal storage diseases, more complex and widespread conditions like Alzheimer’s disease, Parkinson’s disease, epilepsy or chronic pain are increasingly targeted for gene therapy. Increasing numbers of applications and patients to be treated will require improving and simplifying gene therapy protocols to make them accessible to the largest number of affected people. While vectors and manufacturing are a major field of academic research and industrial development, there is a growing need to improve, standardize and simplify delivery methods. Delivery is the major issue for CNS therapies in general, and particularly for gene therapy. The blood brain barrier restricts the passage of vectors; and strategies to bypass this obstacle are a central focus of research. Here, we present the different ways that can be used to deliver gene therapy products to the CNS. We focus on results obtained in large animals that have allowed the transfer of protocols to human patients and have resulted in the generation of clinical data. We discuss the different routes of administration, their advantages and their limitations. We describe techniques, equipment and protocols and how they should be selected for safe delivery and improved efficiency for the next generation of gene therapy trials for CNS diseases.
Autres auteursVoir la publication
Projets
-
Hacking Health Camp 2023 : Projet #39 - Salvia
-
🏆 Prix SML, délivré par le Dr. Thierry Ressel du Syndicat des Médecins Libéraux
🚀 Hacking Health Camp #10 - 24 au 26 mars 2023 : hackathon de 50h pour développer un prototype sur un projet d'innovation en santé
💊 Projet #39 - Salvia : application web qui facilite la prise de décision pour la prescription et la délivrance de médicaments
⚙️ Participation en tant que pharmacien et néo-entrepreneur
🎙️ Pitch final : https://www.youtube.com/watch?v=HApuGeCQ9BU&t=3595sAutres créateursVoir le projet
Prix et distinctions
-
Scholarship : Initiative of Excellence (IdEx)
Strasbourg University, France
6-month funded research internship in fundamental Neuroscience in Montreal
-
Scholarship : BMI Grand Est
Grand Est region, France
6-month funded research internship in fundamental Neuroscience in Montreal
Langues
-
French
Bilingue ou langue natale
-
English
Capacité professionnelle complète
-
German
Compétence professionnelle limitée
-
Spanish
Notions
Recommandations reçues
-
Membre LinkedIn
6 personnes ont recommandé Raphael
Inscrivez-vous pour y accéderAutres profils similaires
Autres personnes nommées Raphael Schatz 🧡
-
Raphael Schatz
-
Raphael Schatz
Field Service Engineer bei Roche Diagnostics Austria
-
Raphael Schatzmann
Architecting Integration - let's build bridges and integrate solutions to empower growth and turn complexity into connectivity
-
Raphael Schatz
Student at Internationale Hochschule IU
6 autres personnes nommées Raphael Schatz 🧡 sont sur LinkedIn
Autres personnes nommées Raphael Schatz 🧡