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| 2. |
- Dahl, Maria, et al.
(författare)
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Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector
- 2021
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Ingår i: Molecular Therapy - Methods and Clinical Development. - : Elsevier BV. - 2329-0501. ; 20, s. 312-323
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Tidskriftsartikel (refereegranskat)abstract
- This study evaluates a clinically applicable lentiviral vector for treatment of Gaucher disease type 1. Hematopoietic stem cells transduced with the vector and transplanted into a mouse model successfully halted or reversed pathology. These data were used as proof-of-concept for regulatory filing enabling the commencement of an international phase 1/2 clinical trial.
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| 3. |
- Dimitriadis, Georgios, et al.
(författare)
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SN 2021zny : an early flux excess combined with late-time oxygen emission suggests a double white dwarf merger event
- 2023
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Ingår i: Monthly notices of the Royal Astronomical Society. - : Oxford University Press (OUP). - 0035-8711 .- 1365-2966. ; 521:1, s. 1162-1183
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Tidskriftsartikel (refereegranskat)abstract
- We present a photometric and spectroscopic analysis of the ultraluminous and slowly evolving 03fg-like Type Ia SN 2021zny. Our observational campaign starts from similar to 5.3 h after explosion (making SN 2021zny one of the earliest observed members of its class), with dense multiwavelength coverage from a variety of ground-and space-based telescopes, and is concluded with a nebular spectrum similar to 10 months after peak brightness. SN 2021zny displayed several characteristics of its class, such as the peak brightness (M-B = -19.95 mag), the slow decline (delta m(15)(B) = 0.62 mag), the blue early-time colours, the low ejecta velocities, and the presence of significant unburned material above the photosphere. However, a fluxexcess for the first similar to 1.5 d after explosion is observed in four photometric bands, making SN 2021zny the third 03fg-like event with this distinct behaviour, while its + 313 d spectrum shows prominent [OI] lines, a very unusual characteristic of thermonuclear SNe. The early flux excess can be explained as the outcome of the interaction of the ejecta with similar to 0 . 04 M-? of H/He-poor circumstellar material at a distance of similar to 10(12) cm, while the low ionization state of the late-time spectrum re veals lo w abundances of stable iron-peak elements. All our observations are in accordance with a progenitor system of two carbon/oxygen white dwarfs that undergo a merger event, with the disrupted white dwarf ejecting carbon-rich circumstellar material prior to the primary white dwarf detonation.
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| 4. |
- Klionsky, Daniel J., et al.
(författare)
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Guidelines for the use and interpretation of assays for monitoring autophagy
- 2012
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Ingår i: Autophagy. - : Informa UK Limited. - 1554-8635 .- 1554-8627. ; 8:4, s. 445-544
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Forskningsöversikt (refereegranskat)abstract
- In 2008 we published the first set of guidelines for standardizing research in autophagy. Since then, research on this topic has continued to accelerate, and many new scientists have entered the field. Our knowledge base and relevant new technologies have also been expanding. Accordingly, it is important to update these guidelines for monitoring autophagy in different organisms. Various reviews have described the range of assays that have been used for this purpose. Nevertheless, there continues to be confusion regarding acceptable methods to measure autophagy, especially in multicellular eukaryotes. A key point that needs to be emphasized is that there is a difference between measurements that monitor the numbers or volume of autophagic elements (e.g., autophagosomes or autolysosomes) at any stage of the autophagic process vs. those that measure flux through the autophagy pathway (i.e., the complete process); thus, a block in macroautophagy that results in autophagosome accumulation needs to be differentiated from stimuli that result in increased autophagic activity, defined as increased autophagy induction coupled with increased delivery to, and degradation within, lysosomes (in most higher eukaryotes and some protists such as Dictyostelium) or the vacuole (in plants and fungi). In other words, it is especially important that investigators new to the field understand that the appearance of more autophagosomes does not necessarily equate with more autophagy. In fact, in many cases, autophagosomes accumulate because of a block in trafficking to lysosomes without a concomitant change in autophagosome biogenesis, whereas an increase in autolysosomes may reflect a reduction in degradative activity. Here, we present a set of guidelines for the selection and interpretation of methods for use by investigators who aim to examine macroautophagy and related processes, as well as for reviewers who need to provide realistic and reasonable critiques of papers that are focused on these processes. These guidelines are not meant to be a formulaic set of rules, because the appropriate assays depend in part on the question being asked and the system being used. In addition, we emphasize that no individual assay is guaranteed to be the most appropriate one in every situation, and we strongly recommend the use of multiple assays to monitor autophagy. In these guidelines, we consider these various methods of assessing autophagy and what information can, or cannot, be obtained from them. Finally, by discussing the merits and limits of particular autophagy assays, we hope to encourage technical innovation in the field.
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