Κυριακή 6 Νοεμβρίου 2016

MiR-29a Assists in Preventing the Activation of Human Stellate Cells and Promotes Recovery From Liver Fibrosis in Mice



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In This Issue



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Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 Splicing



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Research Highlights



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Writing the Next Chapter for the Molecular Therapy Family of Journals



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Preliminary Reports of Stereotaxic Stem Cell Transplants in Chronic Stroke Patients



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Strand and Cell Type-specific Function of microRNA-126 in Angiogenesis



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Boosting Central Nervous System Axon Regeneration by Circumventing Limitations of Natural Cytokine Signaling



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Lentiviral Vector Gene Therapy Protects XCGD Mice From Acute Staphylococcus aureus Pneumonia and Inflammatory Response



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The TMSB4 Pseudogene LncRNA Functions as a Competing Endogenous RNA to Promote Cartilage Degradation in Human Osteoarthritis



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Edible Ginger-derived Nano-lipids Loaded with Doxorubicin as a Novel Drug-delivery Approach for Colon Cancer Therapy



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Targeting of Discoidin Domain Receptor 2 (DDR2) Prevents Myofibroblast Activation and Neovessel Formation During Pulmonary Fibrosis



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Role of HCP5-miR-139-RUNX1 Feedback Loop in Regulating Malignant Behavior of Glioma Cells



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The Retinoid Agonist Tazarotene Promotes Angiogenesis and Wound Healing



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Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing



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Extracellular Antibody Drug Conjugates Exploiting the Proximity of Two Proteins



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Mesenchymal Stromal Cells Mitigate Experimental Colitis via Insulin-like Growth Factor Binding Protein 7-mediated Immunosuppression



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Integrated Safety Assessment of 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human Volunteers



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Corrigendum to “Inducible HGF-secreting Human Umbilical Cord Blood-derived MSCs Produced via TALEN-mediated Genome Editing Promoted Angiogenesis”



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MiR-29a Assists in Preventing the Activation of Human Stellate Cells and Promotes Recovery From Liver Fibrosis in Mice



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In This Issue



http://ift.tt/2eEhel0

Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 Splicing



http://ift.tt/2fwxbaP

Research Highlights



http://ift.tt/2eEjpFn

Writing the Next Chapter for the Molecular Therapy Family of Journals



http://ift.tt/2fwzz12

Preliminary Reports of Stereotaxic Stem Cell Transplants in Chronic Stroke Patients



http://ift.tt/2eEcz2s

Strand and Cell Type-specific Function of microRNA-126 in Angiogenesis



http://ift.tt/2fwwbDf

Boosting Central Nervous System Axon Regeneration by Circumventing Limitations of Natural Cytokine Signaling



http://ift.tt/2eEcxI8

Lentiviral Vector Gene Therapy Protects XCGD Mice From Acute Staphylococcus aureus Pneumonia and Inflammatory Response



http://ift.tt/2fwssFM

The TMSB4 Pseudogene LncRNA Functions as a Competing Endogenous RNA to Promote Cartilage Degradation in Human Osteoarthritis



http://ift.tt/2eEcyvq

Edible Ginger-derived Nano-lipids Loaded with Doxorubicin as a Novel Drug-delivery Approach for Colon Cancer Therapy



http://ift.tt/2fwzyu0

Targeting of Discoidin Domain Receptor 2 (DDR2) Prevents Myofibroblast Activation and Neovessel Formation During Pulmonary Fibrosis



http://ift.tt/2eEjo4h

Role of HCP5-miR-139-RUNX1 Feedback Loop in Regulating Malignant Behavior of Glioma Cells



http://ift.tt/2fwz2Mz

The Retinoid Agonist Tazarotene Promotes Angiogenesis and Wound Healing



http://ift.tt/2eEcziY

Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing



http://ift.tt/2fwuHce

Extracellular Antibody Drug Conjugates Exploiting the Proximity of Two Proteins



http://ift.tt/2eEenIQ

Mesenchymal Stromal Cells Mitigate Experimental Colitis via Insulin-like Growth Factor Binding Protein 7-mediated Immunosuppression



http://ift.tt/2fwuGVI

Integrated Safety Assessment of 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human Volunteers



http://ift.tt/2eEhbpk

Corrigendum to “Inducible HGF-secreting Human Umbilical Cord Blood-derived MSCs Produced via TALEN-mediated Genome Editing Promoted Angiogenesis”



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A Partial Response to Reintroduced Chemotherapy in a Resistant Small Cell Lung Cancer Patient After Priming with RRx-001

As an exceedingly recalcitrant and highly aggressive tumor type without Food and Drug Administration-approved treatment or a known cure, the prognosis of recurrent extensive stage platinum-resistant/refractory small cell lung cancer (SCLC) is worse than other types of lung cancer, and many other tumor types, given a response rate of less than 10% and an overall survival of less than six months. It was broadly classified into three groups based on the initial response to cisplatin/etoposide therapy, platinum-refractory, platinum-resistant, and platinum-sensitive, extensive stage SCLC inevitably relapses, at which point the only standard options are to rechallenge with the first-line chemotherapeutic regimen in the case of sensitive disease or to start the topoisomerase I inhibitor, topotecan. Sensitive disease is defined by a response to the first-line therapy and a treatment-free interval of at least 90 days, while the definitions of refractory and resistant disease, respectively, are nonresponse to the first-line treatment or relapse within 90 days. As an important predictor of response to the second-line treatment, the clinical cutoff of three months (or two months in some cases) for resistant and sensitive disease, which along with performance status prognostically separates patients into high- and low-risk categories, dictates subsequent management. This case report presents a resistant SCLC patient enrolled on a Phase II clinical trial called QUADRUPLE THREAT (formerly TRIPLE THREAT; NCT02489903) who responded to reintroduced platinum doublets after sequential priming with the resistance-reversing epi-immunotherapeutic agent, RRx-001. In the QUADRUPLE THREAT clinical trial, both during priming with RRx-001 and during sequential treatment with platinum doublets, the patient maintained a good quality of life and performance status.

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Giant Coronary Artery Aneurysm Causing Acute Anterior Myocardial Infarction

A 70-year-old man with hypertension was admitted to our coronary ICU with acute anterior MI. Emergent primary PCI was planned and coronary angiography was performed. LAD artery was totally occluded in the proximal segment just after a huge 32 × 26 mm sized aneurysm. Emergent CABG operation was performed in 75 minutes because of multivessel disease including the RCA and left circumflex artery. Aneurysm was ligated and coronary bypass was performed using LIMA and saphenous grafts. The postoperative course of the patient was uneventful. He was discharged with medical therapy including ASA, clopidogrel, and atorvastatin. He was asymptomatic at his polyclinic visit in the first month.

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Becker’s Nevus Syndrome in a Pediatric Female Patient

Becker's nevus syndrome is part of the epidermal nevus syndromes and has been described with a phenotype that includes Becker's nevus, ipsilateral breast hypoplasia, and variable skeletal malformations. It is more frequent in males than in females (5 : 1) but is more relevant in females. The diagnosis is clinically based and the skin lesion must be present and no other numbered criteria have been established, but with more criteria being present the possibility of the diagnosis is higher. Regarding the treatment of breast hypoplasia, the use of antiandrogen medication has demonstrated adequate clinical response in a dose of 50 mg/day of spironolactone.

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A Teenage Girl with Acute Dyspnea and Hypoxemia during Red Blood Cell Transfusion

Transfusion-related acute lung injury (TRALI) can cause morbidity and mortality. We present the case of teenager who developed dyspnea and hypoxemia few hours after red cell transfusion. After being admitted for close monitoring and oxygen therapy, her symptoms spontaneously resolved. Message: dyspnea during red cell transfusion should raise the suspicion of TRALI.

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Lung Ultrasound in Early Diagnosis of Neonatal Ventilator Associated Pneumonia before Any Radiographic or Laboratory Changes

Neonatal pneumonia is reported to be the primary cause of neonatal respiratory failure and one of the common causes of neonatal hospitalization and death in developing countries. Chest X-ray was considered the gold standard for diagnosis of neonatal pneumonia. Lung ultrasonography has been described as a valuable noninvasive tool for the diagnosis of many neonatal pulmonary diseases. We report a case of ventilation associated neonatal pneumonia with very early diagnosis using lung ultrasound before any significant radiographic changes in chest X-ray or laboratory findings suggestive of infection.

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Geriatric factors analyses from FFCD 2001-02 phase III study of first-line chemotherapy for elderly metastatic colorectal cancer patients

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Publication date: Available online 5 November 2016
Source:European Journal of Cancer
Author(s): Thomas Aparicio, Dany Gargot, Laurent Teillet, Emilie Maillard, Dominique Genet, Jacques Cretin, Christophe Locher, Olivier Bouché, Gilles Breysacher, Jean-François Seitz, Mohamed Gasmi, Laetitia Stefani, Mohamed Ramdani, Thierry Lecomte, Dominique Auby, Roger Faroux, Jean-Baptiste Bachet, Céline Lepère, Faiza Khemissa, Iradj Sobhani, Olivier Boulat, Emmanuel Mitry, Jean-Louis Jouve
AimSeveral predictors of metastatic colorectal cancer (mCRC) outcomes have been described. Specific geriatric characteristics could be of interest to determine prognosis.MethodElderly patients (75+) with previously untreated mCRC were randomly assigned to receive infusional 5-fluorouracil-based chemotherapy, either alone (FU) or in combination with irinotecan (IRI). Geriatric evaluations were included as an optional procedure. The predictive value of geriatric parameters was determined for the objective response rate (ORR), progression-free survival (PFS) and overall survival (OS).ResultsFrom June 2003 to May 2010, the FFCD 2001-02 randomised trial enrolled 282 patients. A baseline geriatric evaluation was done in 123 patients; 62 allocated to the FU arm and 61 to the IRI arm. The baseline Charlson index was ≤1 in 75%, Mini-Mental State Examination was ≤27/30 in 31%, Geriatric Depression Scale was >2 in 10% and Instrumental Activities of Daily Living (IADL) was impaired in 34% of the patients. Multivariate analyses revealed that no geriatric parameter was predictive for ORR or PFS. Normal IADL was independently associated with better OS.The benefit of doublet chemotherapy on PFS differed in subgroups of patients ≤80 years, with unresected primary tumour, leucocytes >11,000 mm3 and carcinoembryonic antigen >2N. There was a trend towards better OS in patients with normal IADL.ConclusionThe autonomy score was an independent predictor for OS. A trend toward a better efficacy of doublet chemotherapy in some subgroups of patients was reported and should be further explored.



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Septooptic Dysplasia with an Associated Arachnoid Cyst

A 4-week-old male infant presented with hypothermia, hypoglycemia, and hyperbilirubinemia. His medical history was remarkable for hydrocephalus secondary to an arachnoid cyst, intermittent hypoglycemia, hypothermia, and poor feeding requiring nasogastric tube for nutrition. Physical exam revealed retrognathia, mild hypotonia, micropenis, and clinodactyly. Ophthalmologic exam demonstrated bilateral optic nerve hypoplasia (ONH). Laboratory data confirmed inadequate cortisol and growth hormone response to hypoglycemia, a low thyroxine level, and direct hyperbilirubinemia. Magnetic resonance imaging of the brain confirmed the known history of arachnoid cyst with hydrocephalus but also revealed anterior pituitary hypoplasia, absence of the posterior pituitary bright spot, a thin pituitary stalk, and bilateral optic nerve hypoplasia. A diagnosis of septooptic dysplasia (SOD) was made. Hormone replacement with hydrocortisone and levothyroxine was started with improvement in the infant's glycemic control, thermoregulation, feeding, and cholestasis. This case reinforces the importance of careful physical examination and laboratory review in a patient with known history of arachnoid cyst which has been previously described as an associated feature of optic nerve hypoplasia and hypopituitarism.

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Successful treatment with gefitinib after Stevens–Johnson syndrome associated with afatinib therapy in a patient with adenocarcinoma of the lung

Abstract

We report a case of a 65-year-old woman with stage IV lung adenocarcinoma who experienced Stevens–Johnson syndrome (SJS) during afatinib therapy. The patient received afatinib as the first-line therapy after the confirmation of harboring an exon 19 deletion mutation in the epidermal growth factor receptor (EGFR) gene. The patient presented with multiple erythematous papules mainly on the body trunk and thigh 32 days after afatinib administration. Subsequently, diffuse erosions of oral mucosa and purpuric macules with flat atypical targets emerged. Skin biopsy specimen showed the histology compatible with epidermal necrosis and the patient was diagnosed as having SJS. The symptoms of SJS were recovered by systemic steroid and immunoglobulin treatment. Gefitinib was administered as the third-line therapy after the second-line therapy with carboplatin plus pemetrexed had failed. Tumor shrinkage was obtained shortly and has been maintained without the recurrence of SJS. Rechallenge of tyrosine kinase inhibitor by gefitinib could be an alternative treatment option in patients who experienced SJS by afatinib.



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Successful treatment with gefitinib after Stevens–Johnson syndrome associated with afatinib therapy in a patient with adenocarcinoma of the lung

Abstract

We report a case of a 65-year-old woman with stage IV lung adenocarcinoma who experienced Stevens–Johnson syndrome (SJS) during afatinib therapy. The patient received afatinib as the first-line therapy after the confirmation of harboring an exon 19 deletion mutation in the epidermal growth factor receptor (EGFR) gene. The patient presented with multiple erythematous papules mainly on the body trunk and thigh 32 days after afatinib administration. Subsequently, diffuse erosions of oral mucosa and purpuric macules with flat atypical targets emerged. Skin biopsy specimen showed the histology compatible with epidermal necrosis and the patient was diagnosed as having SJS. The symptoms of SJS were recovered by systemic steroid and immunoglobulin treatment. Gefitinib was administered as the third-line therapy after the second-line therapy with carboplatin plus pemetrexed had failed. Tumor shrinkage was obtained shortly and has been maintained without the recurrence of SJS. Rechallenge of tyrosine kinase inhibitor by gefitinib could be an alternative treatment option in patients who experienced SJS by afatinib.



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