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  1. Article ; Online: Improving access to gene therapy for rare diseases.

    Fox, Thomas A / Booth, Claire

    Disease models & mechanisms

    2024  Volume 17, Issue 6

    Abstract: Effective gene therapy approaches have been developed for many rare diseases, including inborn errors of immunity and metabolism, haemoglobinopathies and inherited blindness. Despite successful pre-clinical and clinical results, these gene therapies are ... ...

    Abstract Effective gene therapy approaches have been developed for many rare diseases, including inborn errors of immunity and metabolism, haemoglobinopathies and inherited blindness. Despite successful pre-clinical and clinical results, these gene therapies are not widely available, primarily for non-medical reasons. Lack of commercial interest in therapies for ultra-rare diseases, costs of development and complex manufacturing processes required for advanced therapy medicinal products (ATMPs) are some of the main problems that are restricting access. The complexities and costs of navigating the regulatory environments in different jurisdictions for treatments that affect small numbers of patients is a problem unique to ATMPS for rare and ultra-rare diseases. In this Perspective, we outline some of the challenges and potential solutions that, we hope, will improve access to gene therapy for rare diseases.
    MeSH term(s) Humans ; Rare Diseases/genetics ; Rare Diseases/therapy ; Genetic Therapy/methods
    Language English
    Publishing date 2024-04-19
    Publishing country England
    Document type Journal Article
    ZDB-ID 2451104-3
    ISSN 1754-8411 ; 1754-8403
    ISSN (online) 1754-8411
    ISSN 1754-8403
    DOI 10.1242/dmm.050623
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article ; Online: Passive transfer - is it time to reset our goals?

    Lindley, George / Booth, Richard / Wathes, Claire / Blackie, Nicola

    The Veterinary record

    2023  Volume 192, Issue 5, Page(s) 216

    Language English
    Publishing date 2023-02-21
    Publishing country England
    Document type Journal Article
    ZDB-ID 390015-0
    ISSN 2042-7670 ; 0042-4900
    ISSN (online) 2042-7670
    ISSN 0042-4900
    DOI 10.1002/vetr.2813
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

    Zs.B 645: Show issues Location:
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  3. Article ; Online: Realizing the Potential of Gene Therapies for Rare and Ultra-Rare Inherited Diseases.

    Booth, Claire / Aiuti, Alessandro

    Human gene therapy

    2023  Volume 34, Issue 17-18, Page(s) 776–781

    Abstract: Rare and ultrarare diseases have been central to the field of gene therapy since its earliest stage, and we are now witnessing more and more effective treatments entering the clinical realm for patients in need. However, despite promising results across ... ...

    Abstract Rare and ultrarare diseases have been central to the field of gene therapy since its earliest stage, and we are now witnessing more and more effective treatments entering the clinical realm for patients in need. However, despite promising results across a range of rare diseases, transformative gene therapies may not be available and accessible to patients for nonmedical reasons. Traditional regulatory and commercialization pathways to licensed products seem to be prohibitive for ultrasmall patient populations. Here, we highlight some of the challenges of delivering gene therapies in rare diseases and discuss innovative solutions being proposed by the gene therapy community.
    MeSH term(s) Humans ; Rare Diseases/genetics ; Rare Diseases/therapy ; Genetic Therapy
    Language English
    Publishing date 2023-08-25
    Publishing country United States
    Document type Journal Article ; Review ; Research Support, Non-U.S. Gov't
    ZDB-ID 1028152-6
    ISSN 1557-7422 ; 1043-0342
    ISSN (online) 1557-7422
    ISSN 1043-0342
    DOI 10.1089/hum.2023.127
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    In stock of ZB MED Cologne/Königswinter

    Zs.A 2988: Show issues Location:
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  4. Article ; Online: Food and nutrition information requirements of Australian primary school parents.

    Aydin, Gozde / Margerison, Claire / Worsley, Anthony / Booth, Alison

    Public health nutrition

    2024  Volume 27, Issue 1, Page(s) e65

    Abstract: Objective: To explore what Australian primary school parents want to learn about food and nutrition to improve their children's eating behaviours, as well as the associations between parents' personal and demographic characteristics and their views ... ...

    Abstract Objective: To explore what Australian primary school parents want to learn about food and nutrition to improve their children's eating behaviours, as well as the associations between parents' personal and demographic characteristics and their views regarding their food and nutrition knowledge needs.
    Design: An online nationwide cross-sectional survey was conducted in 2021 using a mixed-methods approach. Logistic regression analysis was utilised to examine the relationship between parents' demographics, personal values and their views. Content analysis was performed using Leximancer.
    Setting: Australia.
    Participants: Seven hundred and eighty-seven parents.
    Results: Fifty-one per cent wanted to learn more about food and nutrition to improve their children's healthy eating habits, and 77% of those preferred schools to provide that information. Online/printed newsletters and YouTube were the most preferred methods for receiving food and nutrition related information. Higher universalism-concern value (concern for the welfare of those in the larger society and world) scores were positively associated parents' preference for schools to provide food and nutrition-related information. Parents with non-English-speaking backgrounds and younger parents were more likely to want to learn about food and nutrition. Parents wanted to learn more about encouraging healthy eating, ideas for the lunchbox, food labels and age-specific portion sizes and recommendations.
    Conclusions: Findings can inform public health educators and assist them in designing future food and nutrition education programmes and resources targeting primary school parents.
    MeSH term(s) Child ; Humans ; Australia ; Cross-Sectional Studies ; Parents ; Food ; Feeding Behavior ; Schools
    Language English
    Publishing date 2024-02-05
    Publishing country England
    Document type Journal Article
    ZDB-ID 1436024-x
    ISSN 1475-2727 ; 1368-9800
    ISSN (online) 1475-2727
    ISSN 1368-9800
    DOI 10.1017/S1368980024000387
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  5. Article ; Online: What a Clinician Needs to Know About Genome Editing: Status and Opportunities for Inborn Errors of Immunity.

    Mudde, Anne C A / Kuo, Caroline Y / Kohn, Donald B / Booth, Claire

    The journal of allergy and clinical immunology. In practice

    2024  

    Abstract: During the past 20 years, gene editing has emerged as a novel form of gene therapy. Since the publication of the first potentially therapeutic gene editing platform for genetic disorders, increasingly sophisticated editing technologies have been ... ...

    Abstract During the past 20 years, gene editing has emerged as a novel form of gene therapy. Since the publication of the first potentially therapeutic gene editing platform for genetic disorders, increasingly sophisticated editing technologies have been developed. As with viral vector-mediated gene addition, inborn errors of immunity are excellent candidate diseases for a corrective autologous hematopoietic stem cell gene editing strategy. Research on gene editing for inborn errors of immunity is still entirely preclinical, with no trials yet underway. However, with editing techniques maturing, scientists are investigating this novel form of gene therapy in context of an increasing number of inborn errors of immunity. Here, we present an overview of these studies and the recent progress moving these technologies closer to clinical benefit.
    Language English
    Publishing date 2024-01-19
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2843237-X
    ISSN 2213-2201 ; 2213-2198
    ISSN (online) 2213-2201
    ISSN 2213-2198
    DOI 10.1016/j.jaip.2024.01.019
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  6. Article ; Online: Gene therapy for inborn error of immunity - current status and future perspectives.

    Mudde, Anne / Booth, Claire

    Current opinion in allergy and clinical immunology

    2022  Volume 23, Issue 1, Page(s) 51–62

    Abstract: Purpose of review: Development of hematopoietic stem cell (HSC) gene therapy (GT) for inborn errors of immunity (IEIs) continues to progress rapidly. Although more patients are being treated with HSC GT based on viral vector mediated gene addition, gene ...

    Abstract Purpose of review: Development of hematopoietic stem cell (HSC) gene therapy (GT) for inborn errors of immunity (IEIs) continues to progress rapidly. Although more patients are being treated with HSC GT based on viral vector mediated gene addition, gene editing techniques provide a promising new approach, in which transgene expression remains under the control of endogenous regulatory elements.
    Recent findings: Many gene therapy clinical trials are being conducted and evidence showing that HSC GT through viral vector mediated gene addition is a successful and safe curative treatment option for various IEIs is accumulating. Gene editing techniques for gene correction are, on the other hand, not in clinical use yet, despite rapid developments during the past decade. Current studies are focussing on improving rates of targeted integration, while preserving the primitive HSC population, which is essential for future clinical translation.
    Summary: As HSC GT is becoming available for more diseases, novel developments should focus on improving availability while reducing costs of the treatment. Continued follow up of treated patients is essential for providing information about long-term safety and efficacy. Editing techniques have great potential but need to be improved further before the translation to clinical studies can happen.
    MeSH term(s) Humans ; Genetic Therapy/methods ; Gene Editing/methods ; Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells/metabolism ; Genetic Vectors/genetics
    Language English
    Publishing date 2022-12-06
    Publishing country United States
    Document type Review ; Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 2088710-3
    ISSN 1473-6322 ; 1528-4050
    ISSN (online) 1473-6322
    ISSN 1528-4050
    DOI 10.1097/ACI.0000000000000876
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    Zs.A 5666: Show issues Location:
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  7. Article ; Online: Parental support for free school lunches in Australian primary schools: associated factors and perceived barriers.

    Aydin, Gozde / Margerison, Claire / Worsley, Anthony / Booth, Alison

    Public health nutrition

    2023  Volume 26, Issue 12, Page(s) 3320–3330

    Abstract: Objective: (1) To explore the feasibility of such programmes in Australia, this study examined parents' views on free school lunch provision. (2) To examine the associations between parents' demographic and personal characteristics and their support for ...

    Abstract Objective: (1) To explore the feasibility of such programmes in Australia, this study examined parents' views on free school lunch provision. (2) To examine the associations between parents' demographic and personal characteristics and their support for free universal school lunches.
    Design: An online cross-sectional survey of parents.
    Setting: Australia, April 2021.
    Participants: Seven hundred and eighty-seven parents took the survey. They had a mean age of 40. The respondents were predominantly female (95 %) and had a university degree (72 %).
    Results: Fifty-three percentage of the respondents agreed that all students should have access to healthy and well-balanced, free school lunches. Parents were concerned about healthiness, catering, allergies and cost of school-provided school lunches. Ethnic background, universalism values and education levels were significantly associated with support for free school lunch provision. Non-native English-speaking parents were almost three times more likely to support free universal lunches in primary schools than their native English-speaking counterparts. Parents with higher universalism-concern values were more likely to endorse free lunches in primary school. However, the level of education was negatively associated with parents' support for free school lunches.
    Conclusions: The survey results highlight the complexity of parental views on free school lunch provision. Parents' concerns regarding lunches should be considered in developing school lunch programmes that meet the needs and preferences of diverse communities. These findings can be used to guide future primary school lunch provision initiatives.
    MeSH term(s) Humans ; Female ; Adult ; Male ; Lunch ; Cross-Sectional Studies ; Australia ; Schools ; Parents ; Food Services
    Language English
    Publishing date 2023-10-26
    Publishing country England
    Document type Journal Article
    ZDB-ID 1436024-x
    ISSN 1475-2727 ; 1368-9800
    ISSN (online) 1475-2727
    ISSN 1368-9800
    DOI 10.1017/S1368980023002240
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Bonn / Germany

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  8. Article ; Online: Gene Therapy for Inborn Errors of Immunity.

    Arlabosse, Tiphaine / Booth, Claire / Candotti, Fabio

    The journal of allergy and clinical immunology. In practice

    2023  Volume 11, Issue 6, Page(s) 1592–1601

    Abstract: In the early 1990s, gene therapy (GT) entered the clinical arena as an alternative to hematopoietic stem cell transplantation for forms of inborn errors of immunity (IEIs) that are not medically manageable because of their severity. In principle, the use ...

    Abstract In the early 1990s, gene therapy (GT) entered the clinical arena as an alternative to hematopoietic stem cell transplantation for forms of inborn errors of immunity (IEIs) that are not medically manageable because of their severity. In principle, the use of gene-corrected autologous hematopoietic stem cells presents several advantages over hematopoietic stem cell transplantation, including making donor searches unnecessary and avoiding the risks for graft-versus-host disease. In the past 30 years or more of clinical experience, the field has witnessed multiple examples of successful applications of GT to a number of IEIs, as well as some serious drawbacks, which have highlighted the potential genotoxicity of integrating viral vectors and stimulated important progress in the development of safer gene transfer tools. The advent of gene editing technologies promises to expand the spectrum of IEIs amenable to GT to conditions caused by mutated genes that require the precise regulation of expression or by dominant-negative variants. Here, we review the main concepts of GT as it applies to IEIs and the clinical results obtained to date. We also describe the challenges faced by this branch of medicine, which operates in the unprofitable sector of human rare diseases.
    MeSH term(s) Humans ; Genetic Therapy/methods ; Hematopoietic Stem Cell Transplantation/methods ; Graft vs Host Disease
    Language English
    Publishing date 2023-04-20
    Publishing country United States
    Document type Review ; Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 2843237-X
    ISSN 2213-2201 ; 2213-2198
    ISSN (online) 2213-2201
    ISSN 2213-2198
    DOI 10.1016/j.jaip.2023.04.001
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 7086: Show issues Location:
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  9. Article ; Online: Parental support for free school lunches in Australian primary schools

    Gozde Aydin / Claire Margerison / Anthony Worsley / Alison Booth

    Public Health Nutrition, Vol 26, Pp 3320-

    associated factors and perceived barriers

    2023  Volume 3330

    Abstract: Abstract Objective: (1) To explore the feasibility of such programmes in Australia, this study examined parents’ views on free school lunch provision. (2) To examine the associations between parents’ demographic and personal characteristics and their ... ...

    Abstract Abstract Objective: (1) To explore the feasibility of such programmes in Australia, this study examined parents’ views on free school lunch provision. (2) To examine the associations between parents’ demographic and personal characteristics and their support for free universal school lunches. Design: An online cross-sectional survey of parents. Setting: Australia, April 2021. Participants: Seven hundred and eighty-seven parents took the survey. They had a mean age of 40. The respondents were predominantly female (95 %) and had a university degree (72 %). Results: Fifty-three percentage of the respondents agreed that all students should have access to healthy and well-balanced, free school lunches. Parents were concerned about healthiness, catering, allergies and cost of school-provided school lunches. Ethnic background, universalism values and education levels were significantly associated with support for free school lunch provision. Non-native English-speaking parents were almost three times more likely to support free universal lunches in primary schools than their native English-speaking counterparts. Parents with higher universalism-concern values were more likely to endorse free lunches in primary school. However, the level of education was negatively associated with parents’ support for free school lunches. Conclusions: The survey results highlight the complexity of parental views on free school lunch provision. Parents’ concerns regarding lunches should be considered in developing school lunch programmes that meet the needs and preferences of diverse communities. These findings can be used to guide future primary school lunch provision initiatives.
    Keywords School lunch ; Child nutrition ; Health promotion ; Primary school ; Food services ; Public aspects of medicine ; RA1-1270 ; Nutritional diseases. Deficiency diseases ; RC620-627
    Subject code 370
    Language English
    Publishing date 2023-12-01T00:00:00Z
    Publisher Cambridge University Press
    Document type Article ; Online
    Database BASE - Bielefeld Academic Search Engine (life sciences selection)

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  10. Article: Parents' Communication with Teachers about Food and Nutrition Issues of Primary School Students.

    Aydin, Gozde / Margerison, Claire / Worsley, Anthony / Booth, Alison

    Children (Basel, Switzerland)

    2022  Volume 9, Issue 4

    Abstract: Parents and teachers have a major influence in the formation of primary school children's eating behaviours. Although the cooperation of parents and teachers has frequently been recommended in the promotion of healthy eating habits among primary school ... ...

    Abstract Parents and teachers have a major influence in the formation of primary school children's eating behaviours. Although the cooperation of parents and teachers has frequently been recommended in the promotion of healthy eating habits among primary school children, little is known about the communication between these two groups regarding food- and nutrition-related issues. This paper reports findings from semi-structured interviews with primary school parents (
    Language English
    Publishing date 2022-04-04
    Publishing country Switzerland
    Document type Journal Article
    ZDB-ID 2732685-8
    ISSN 2227-9067
    ISSN 2227-9067
    DOI 10.3390/children9040510
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