scholarly journals A Virtual Reality Orientation and Mobility Test for Inherited Retinal Degenerations: Testing a Proof-of-Concept After Gene Therapy

2021 ◽  
Vol Volume 15 ◽  
pp. 939-952
Author(s):  
Tomas S Aleman ◽  
Alexander J Miller ◽  
Katherine H Maguire ◽  
Elena M Aleman ◽  
Leona W Serrano ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Virtual Reality ◽  
Proof Of Concept ◽  
Retinal Degenerations ◽  
Mobility Test ◽  
Orientation And Mobility ◽  
Reality Orientation

scholarly journals Enhancing the Optical Efficiency of Near-Eye Displays with Liquid Crystal Optics

Crystals ◽  
2021 ◽  
Vol 11 (2) ◽  
pp. 107
Author(s):  
Tao Zhan ◽  
En-Lin Hsiang ◽  
Kun Li ◽  
Shin-Tson Wu
Keyword(s):  
Virtual Reality ◽  
Liquid Crystal ◽  
High Efficiency ◽  
Berry Phase ◽  
Optical Element ◽  
Liquid Crystal Polymer ◽  
Proof Of Concept ◽  
Optical Efficiency ◽  
Crystal Polymer ◽  
Light Efficiency

We demonstrate a light efficient virtual reality (VR) near-eye display (NED) design based on a directional display panel and a diffractive deflection film (DDF). The DDF was essentially a high-efficiency Pancharatnam-Berry phase optical element made of liquid crystal polymer. The essence of this design is directing most of the display light into the eyebox. The proposed method is applicable for both catadioptric and dioptric VR lenses. A proof-of-concept experiment was conducted with off-the-shelf optical parts, where the light efficiency was enhanced by more than 2 times.

scholarly journals Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side

Pharmaceutics ◽  
2020 ◽  
Vol 12 (6) ◽  
pp. 549
Author(s):  
Laura Garcia-Perez ◽  
Anita Ordas ◽  
Kirsten Canté-Barrett ◽  
Pauline Meij ◽  
Karin Pike-Overzet ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Severe Combined Immunodeficiency ◽  
Good Manufacturing Practice ◽  
Proof Of Concept ◽  
Preclinical Development ◽  
Hematopoietic Stem ◽  
Immune Deficiencies ◽  
Suitable Vector

Recent clinical trials using patient’s own corrected hematopoietic stem cells (HSCs), such as for primary immunodeficiencies (Adenosine deaminase (ADA) deficiency, X-linked Severe Combined Immunodeficiency (SCID), X-linked chronic granulomatous disease (CGD), Wiskott–Aldrich Syndrome (WAS)), have yielded promising results in the clinic; endorsing gene therapy to become standard therapy for a number of diseases. However, the journey to achieve such a successful therapy is not easy, and several challenges have to be overcome. In this review, we will address several different challenges in the development of gene therapy for immune deficiencies using our own experience with Recombinase-activating gene 1 (RAG1) SCID as an example. We will discuss product development (targeting of the therapeutic cells and choice of a suitable vector and delivery method), the proof-of-concept (in vitro and in vivo efficacy, toxicology, and safety), and the final release steps to the clinic (scaling up, good manufacturing practice (GMP) procedures/protocols and regulatory hurdles).

scholarly journals Developing an Outcome Measure With High Luminance for Optogenetics Treatment of Severe Retinal Degenerations and for Gene Therapy of Cone Diseases

2016 ◽  
Vol 57 (7) ◽  
pp. 3211 ◽  
Author(s):  
Artur V. Cideciyan ◽  
Alejandro J. Roman ◽  
Samuel G. Jacobson ◽  
Boyuan Yan ◽  
Michele Pascolini ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Outcome Measure ◽  
High Luminance ◽  
Retinal Degenerations

Improving Cognitive and Emotional State Using 3D Virtual Reality Orientation Game

2020 ◽  
pp. 220-225
Author(s):  
Manish Kumar Jha ◽  
Marwa Boukadida ◽  
Hamdi Ben Abdessalem ◽  
Alexie Byrns ◽  
Marc Cuesta ◽  
...  
Keyword(s):  
Virtual Reality ◽  
Emotional State ◽  
Reality Orientation

scholarly journals Hemophilia gene therapy: ushering in a new treatment paradigm?

Hematology ◽  
2021 ◽  
Vol 2021 (1) ◽  
pp. 226-233
Author(s):  
Lindsey A. George
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Clinical Trials ◽  
Hemophilia A ◽  
Full Potential ◽  
Proof Of Concept ◽  
Aav Vector ◽  
Treatment Paradigm ◽  
Clinical Trial Enrollment ◽  
New Treatment

Abstract After 3 decades of clinical trials, repeated proof-of-concept success has now been demonstrated in hemophilia A and B gene therapy. Current clinical hemophilia gene therapy efforts are largely focused on the use of systemically administered recombinant adeno-associated viral (rAAV) vectors for F8 or F9 gene addition. With multiple ongoing trials, including licensing studies in hemophilia A and B, many are cautiously optimistic that the first AAV vectors will obtain regulatory approval within approximately 1 year. While supported optimism suggests that the goal of gene therapy to alter the paradigm of hemophilia care may soon be realized, a number of outstanding questions have emerged from clinical trial that are in need of answers to harness the full potential of gene therapy for hemophilia patients. This article reviews the use of AAV vector gene addition approaches for hemophilia A and B, focusing specifically on information to review in the process of obtaining informed consent for hemophilia patients prior to clinical trial enrollment or administering a licensed AAV vector.

scholarly journals Gene Therapy for Retinitis Pigmentosa Caused by MFRP Mutations: Human Phenotype and Preliminary Proof of Concept

2012 ◽  
Vol 23 (4) ◽  
pp. 367-376 ◽  
Author(s):  
Astra Dinculescu ◽  
Jackie Estreicher ◽  
Juan C. Zenteno ◽  
Tomas S. Aleman ◽  
Sharon B. Schwartz ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Retinitis Pigmentosa ◽  
Proof Of Concept ◽  
Human Phenotype
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