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1.
Curr Gene Ther ; 23(3): 184-197, 2023.
Article in English | MEDLINE | ID: mdl-36999706

ABSTRACT

Adeno-associated viruses (AAV) are widely used as a recombinant vectors in gene therapy. AAVs are non-pathogenic. They present reduced cytotoxicity and can transduce both dividing and non-dividing cells. The existence of different serotypes provides flexibility for targeting different tissues and organs. Its therapeutic success was already shown by the approval of three products by the European and American regulatory agencies. To satisfy the high dosage, safety, and reproducibility required in each clinical trial, production platforms based on stable mammalian cell lines have been proposed as the best strategy. However, the methodologies employed must be adapted to each cell line, which often results in distinct productivities. In this article, we review the published and commercially available mammalian stable cell lines, discussing the key factors that impact viral production yields, such as integration sites and copy numbers.


Subject(s)
Dependovirus , Genetic Vectors , Animals , Genetic Vectors/genetics , Dependovirus/genetics , Reproducibility of Results , Cell Line , Mammals/genetics
2.
Galicia clin ; 83(4): 48-50, oct.-dic. 2022. ilus, tab
Article in English | IBECS | ID: ibc-214897

ABSTRACT

Henoch-Schonlein Purpura (HSP) is a systemic vasculitis now called IgA vasculitis, that infrequently occurs in adults. While rheumatological andgastrointestinal complications are common, lung involvement is a rare complication. We report the case of a 70 year-old female that presented tothe hospital with purpuric lesions on her legs and ileitis. During her hospital stay she developed respiratory distress, and the computed tomography (CT scan) showed evidence of Diffuse Alveolar Hemorrhage (DAH). She also had renal dysfunction, and the renal biopsy confirmed the presence of leukocytoclastic vasculitis on histology, consistent with HSP. The patient was initiated on corticosteroid therapy and cyclophosphamide,in addition to supportive management, leading to the resolution of her symptoms.This case is unique, as it presents a rare complication of HSP, with pulmonary disease, causing DAH. Furthermore, adult onset HSP is also anuncommon occurrence.It is very important to recognize DAH early in HSP, as it holds a high mortality rate. (AU)


Subject(s)
Humans , Female , Aged , IgA Vasculitis , Vasculitis , Immunoglobulin A
3.
Eur J Case Rep Intern Med ; 8(11): 002856, 2021.
Article in English | MEDLINE | ID: mdl-34912735

ABSTRACT

Drug-induced liver injury (DILI) is a challenging diagnosis since a wide variety of medicines can cause adverse reactions. Losartan is an angiotensin II receptor antagonist (ARA-II) approved for the treatment of arterial hypertension. The most common adverse effects are fatigue, anaemia, weakness and cough. An increase in transaminases has been reported with less frequency (<2% of cases). Although the mechanism is not fully understood, DILI onset is usually within 1-8 weeks of therapy, and hepatic enzymology usually normalizes 2-4 months after drug suspension. The authors present the case of a 66-year-old male patient with a medical history of arterial hypertension and a prior hospitalization (4 years previously) for drug-induced hepatitis, which, at the time, was attributed to a dietary supplement. Four years later, because of new onset of hypertension, losartan was reintroduced. After 3 weeks, the patient was admitted to the emergency department with complaints of acute abdominal pain associated with asthenia, nausea and increased abdominal volume that had first developed 8 days previously. After exclusion of other causes, DILI associated with losartan was assumed. This is a very rare adverse effect since only seven cases have been described in the literature. LEARNING POINTS: Toxic hepatitis due to losartan is very rare, despite widespread use of the drug.A high index of suspicion for drug-induced liver injury (DILI) should be maintained while other diagnoses are carefully excluded.Monitoring the use of the offending drug is crucial, since DILI recurrence is associated with worse prognosis.

4.
Cureus ; 13(10): e19018, 2021 Oct.
Article in English | MEDLINE | ID: mdl-34853746

ABSTRACT

Q fever is a zoonosis caused by Coxiella burnetii with worldwide distribution at the increasing expression in Europe and endemic in Portugal. It is transmitted by inhalation of aerosols containing spores, main reservoir being cattle, goats and sheep as by ingesting cottage cheese or unpasteurized milk. The majority of patients are asymptomatic; however, they may present with fever, atypical pneumonia, acute hepatitis, cutaneous manifestations and rarely with cardiac or neurological involvement. Although most cases are self-limited, focal persistent or chronic Q fever can manifest years after the onset, wherefore follow-up is essential. The clinical heterogeneity may be so variable that the disease is often diagnosed only if it has been systematically considered. It should be especially taken into account in the presence of risk factors as valvular or joint prostheses, immunocompromised patients, pregnant women and epidemiological setting. The authors present a rare case of Coxiella burnetii pneumonia with cutaneous and hepatic manifestations without any risk factor. This case aims to emphasize the importance of Q fever in the differential diagnosis of fever or atypical pneumonia, even in the absence of known risk factors. The diagnosis is often challenging for clinicians and it is necessary to maintain a high index of suspicion. In Europe and specifically in Portugal is mandatory to report the cases to establish the real impact of this disease.

5.
Galicia clin ; 82(3): 160-161, Julio-Agosto-Septiembre 2021. ilus
Article in English | IBECS | ID: ibc-221614

ABSTRACT

New 2019-Coronavirus (SARS-CoV-2) pandemic is presenting daily diagnostic challenges to emergency departments (ED) doctors, especially as an increasing number of atypical clinical presentations have been reported, including acute sialadenitis (most of parotid). We present a case of a 46-year-old obese caucasian woman, with no relevant medical history, that presented in ED with complaints suggesting parotitis, with normal laboratory tests, at first with no virus isolation, confirmed by computed tomography. One week later, anosmia and dysgeusia were notice, and SARS-CoV-2 was tested and confirmed. Authors want to alert that SARS-CoV-2 should be excluded as parotitis etiology, highly important, especially in this pandemic phase, as a way of transmission control. (AU)


Subject(s)
Humans , Female , Adult , Parotitis , Pandemics , Coronavirus Infections/epidemiology
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