News

New collaborative publication on outcome measures.

Our latest publication “A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures” was recently published.

As it is the case with several of our publications, it summarises a collaboration between different research labs to agree on protocols and cross-validate the methods we use to quantify exon skipping in cell culture, the first step in the screening of new antisense oligonucleotide drugs.

This collaboration was facilitated by COST Action networking grant BM1207 and researchers from our group at Biocruces Bizkaia collaborated with scientists from:

  • Leiden University Medical Center in The Netherlands;
  • University of Ferrara,  Italy;
  • University College London Great Ormond Street Institute of Child Health, United Kingdom;
  • BioMarin Nederland B.V., The Netherlands;
  • Royal Holloway University of London, United Kingdom;
  • University of Northampton, Northampton, United Kingdom
  • Institute of Myology, France

Neurogune Congress

 

Neurogune is the network of the Basque community working for the advance of Neurosciences. We went to the annual congress organized by Neurogune on september 7, 2018, and presented a poster entitled “CRISPR/Cas gene editing in Duchenne muscular dystrophy cultures to test new treatments for the disease”.

 

III Workshop organized by Duchenne Parent Project Spain on the Transfer of Scientific Knowledge in Duchenne muscular dystrophy

 

 

Duchenne Parent Project Spain is a non-profit association created and directed by parents of children with Duchenne and Becker.

On September 7, 2018, we went to the 3rd III Workshop organized by Duchenne Parent Project Spain on the Transfer of Scientific Knowledge in Duchenne muscular dystrophy of the University of Barcelona celebrated the 5th World Day of Duchenne Awareness Duchenne (DMD). It involved more than 45 Spanish scientists who had the opportunity to discuss the research carried out in Spain in Duchenne Muscular Dystrophy, thus establishing future collaborations to advance in the search for effective treatments for DMD.

“The objective of this workshop is to establish communication and collaboration between the different scientists who are investigating the Duchenne and Becker Muscular Dystrophies in Spain, thus facilitating the tools for a rapid advance in these rare diseases” says Dr. Marisol Montolio, Scientific Director of Duchenne Parent Project Spain.

 

World Duchenne Awareness Day

September 7 will be the World Duchenne Awareness Day.

We will be joining many other spanish researchers at the scientific workshop organised by Duchenne Parent Project in Barcelona.

 

 

Dr Sabater-Molina visited us on July 2018

Dr  Sabater Molina visited our lab on July to learn antisense oligonucleotide transfection techniques to be able to apply them in the project awarded to her by The Spanish Society of Cardiology (SEC). This project, in which we participate as collaborators, is titled “antisense oligonucleotides as a new therapeutic strategy in hypertrophic cardiomyopathy” and will be completed at  Hospital de la Arrixaca in Murcia.

ICNMD 2018 | 15th International Congress On Neuromuscular Disease

 

 

 

 

 

The aim of the 15th International Congress On Neuromuscular Disease is to present a wide spectrum of neuromuscular diseases from the perspectives of advances in research, diagnosis and treatment.

Dr Virginia Arechavala and Patricia Soblechero attended the Congress and presented our work on CRISPR/Cas gene editing for in Duchenne muscular dystrophy cultures to test new treatments for the disease and  Myoblots for the evaluation of new treatments in neuromuscular disorders.

XXXI NATIONAL  NEUROMUSCULAR DISORDERS CONGRESS

The 31st National Neuromuscular Disorders Congress was held last 24th and 25th of November in Granada.

Dr Arechavala-Gomeza gave a lecture on the basis of gene therapy, gene editing and RNA therapy that was streamed live and may be played again at Federación ASEM’s facebook page.

 

15th of November, National NMD day

Tomorrow, 15th of November is Neuromuscular Diseases Day in Spain. We joined the campaign, joining hands with other researchers at Biocruces Health Research Institute, in support of research of neuromuscular diseases.

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INPhINIT PhD Fellowship project available.

Our group is in the list of available hosts for the INPhINIT  PhD fellowships program. Candidates can apply to the program and, if selected, apply to our project for a 3 year funded PhD. If you are interested in applying with us, please contact us.

INPhINIT fellows enjoy a 3-year contract under the Spanish labour legislation in force, respecting health and safety, and social security provisions in a stimulating research training environment, with access to appropriate equipment, facilities and opportunities.

In addition to the interdisciplinary training activities offered by the Research Centre, the INPhINIT programme sets up a comprehensive complementary skills training scheme, a mentoring scheme, networking and public engagement activities.

At the time of recruitment, candidates must comply with one of the following options:

To have completed the studies that lead to an official Spanish (or from another country of the European Higher Education Area) university degree awarding 300 ECTS credits, of which at least 60 ECTS credits must correspond to master level.

To have completed a degree in a non-Spanish university not adapted to the European Higher Education Area that gives access to doctoral studies. The verification of an equivalent level of studies to the ones mentioned above will be made by the university when the admission procedure starts.

Candidates must not have resided or carried out their main activity (work, studies, etc.) in Spain for more than 12 months in the 3 years immediately prior to the publication date of the final list of selected candidates (29 may 2018). Short stays such as holidays will not be taken into account when calculating the mobility requirement.

Candidates must have a demonstrable level of English (B2 or higher).

If you want to apply for an INPhINIT fellowship, contact us and click on the link below to create your personal account and fill in the on-line application form.

Go to the on-line application form

New publication: dystrophin quantification in cell culture.

We are happy to announce that we have published our newly developed method to quantify dystrophin and other muscle proteins in cell culture. We expect that this may contribute a faster preclinical development of new treatments for neuromuscular disorders and you may read all about it in Neuropathology and Applied Neurobiology.

“Myoblots: dystrophin quantification by in-cell western assay for a streamlined development of Duchenne muscular dystrophy treatments”. E. Ruiz-Del-Yerro, Garcia-Jimenez I, Arechavala-Gomeza V, Oct 2017 Neuropathol Appl Neurobiol

Summer course on rare diseases (Video in spanish)

Dr. Arechavala took part in a Summer Course on rare diseases last summer and a recording to that seminar can be found here (video in spanish):

https://www.institutoroche.es/jornadas/69-las-enfermedades-raras-un-reto-global-para-el-sistema-sanitario/multimedia/259

 

Looking for pre-doctoral candidates

We are looking for pre-doctoral candidates to apply for funding together. A copy of the ad, in spanish, is below. If you are interested, contact us.

Buscamos candidatos para optar a becas predoctorales de concurso público y realizar una tesis doctoral en el grupo de enfermedades neuromusculares del instituto de investigación sanitaria Biocruces.

El grupo de Enfermedades Neuromusculares centra su interés en la evaluación de nuevos compuestos terapéuticos para estas enfermedades, en particular la distrofia muscular de Duchenne. Actualmente está realizando proyectos para evaluar oligonucleótidos antisentido y técnicas de edición génica CRISPR/Cas. Para más información sobre el grupo, consultar: www.arechavala-lab.com.

Se requiere:

Licenciatura o grado en ciencias de la salud.

Expediente académico superior a 2.5 (escala 1-4) o superior a 8 en escala 1-10.

Estar cursando/haber cursado un Máster Universitario Oficial que le garantice el acceso a un programa de Doctorado en el curso 2016/2017.

Excelentes habilidades comunicativas y organizativas.

Manejo de programas informáticos (Excel, Word, Access, etc).

Idiomas: inglés hablado y escrito (nivel alto).

Disponibilidad para viajar.

Capacidad para trabajar independientemente así como en equipo.

Se valorará, aunque no sea necesario, experiencia en:

Conocimiento y experiencia en técnicas de Biología Molecular: extracción y purificación de DNA, RNA y proteínas, transformación y transfección, PCR.

Experiencia en cultivo celular.

Experiencia en PCR  cuantitativo.

Experiencia en manejo de muestras biológicas.

Experiencia en técnicas de histología: procesamiento de tejidos, fijación e inclusión, tinciones histológicas básicas.

Experiencia en técnicas de inmunohistoquímica e inmunofluorescencia.

Experiencia en el uso de vectores virales.

Conocimiento del manejo de programas estadísticos

Idiomas: euskera hablado y escrito.

Contacto: virginia.arechavalagomeza@osakidetza.eus

EMBO Molecular Biology Publication

Remember when we told you about a new manuscript we were working on? It was published last May in EMBO Molecular Medicine.

It is a review of the delivery hurdles that antisense oligonucleotides face before they can become a good therapeutic option.

You can read it here.

“Delivery is key: lessons learnt from developing splice-switching antisense therapies.”  C. Godfrey, L. R Desviat, B. Smedsrød, F. Piétri-Rouxel, M. A. Denti, P. Disterer, S. Lorain, G. Nogales-Gadea, V. Sardone, R. Anwar, S. El Andaloussi, T. Lehto, B. Khoo, C. Brolin, W M.C.van Roon-Mom, A.Goyenvalle, A. Aartsma-Rus, V. Arechavala-Gomeza

DOI 10.15252/emmm.201607199 | Published online 13.03.2017

EMBO Molecular Medicine (2017) 9, 545-557

Ice Bucket Challenge 2016

 

Another year, another #icebucketchallenge. It did not feel quite as cold, knowing about the recent research results that derived from the funding raised by the original challenge. Still, quite a lot to do, so there we go again.

 

As Dr Arechavala’s PhD was funded by the Motor Neurone Disease Association (MNDA) in the UK, she kindly requests that you consider a donation to either MNDA or FUNDELA, (the Spanish foundation for research in MND).

 

 

Neurogune 2016

We participated in the latest edition of Neurogune, held this time in Bilbao and we had the opportunity to meet many neuroscientist working in the region. Lots of common goals and a very constructive meeting.

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Summer Course on Rare Diseases, San Sebastian.

Dr. Arechavala-Gomeza took part in the Summer Course organised by the University of the Basque Country last 23rd-24th of June titled “Las Enfermedades Raras: un reto global para el sistema sanitaria”, where she could advocate for collaboration and investment in basic scientific research in the development of new therapies for rare diseases.

It was a great opportunity to discuss the current state of the art with a very wide representation of stakeholders in rare diseases and credit should be given to the organisers, Roberto Fernández-Torrón and Adolfo Lopez de Munain, from BioDonostia.

Author's copy of our recent publication available in publications

We have generated an open access version of our recent Lancet Neurology publication, as the original version is behind a paywall. You may access our version at the publications section of this page.

There is also a very interesting podcast associated to this publication, that you may access here:

 

 

Interested in applying to a post-FIR, MIR, BIR, QIR fellowship?

If you finished your residence training recently (2014, 2015 or 2016), you may be able to apply with us to the”Becas BBK/BioCruces” (check last year’s call here: http://becas.bbk.es/cs/Satellite/becasbbk/es/nuestras_becas/becas_de_investigacion ).

They cover a year’s worth of salary (80%) and they are compatible with other short term contracts, so you may still be on call or take short term contracts at the hospital.

If you are interested, contact us.

Looking for a pre-doctoral candidate.

We are looking for candidates to apply for funding together. A copy of the ad, in spanish, is below. If you are interested, contact us.

Buscamos candidatos para optar a becas predoctorales de concurso público y realizar una tesis doctoral en el grupo de enfermedades neuromusculares del instituto de investigación sanitaria BioCruces.

 

El grupo de Enfermedades Neuromusculares centra su interés en la evaluación de nuevos compuestos terapéuticos para estas enfermedades, en particular la distrofia muscular de Duchenne. Actualmente está realizando proyectos para evaluar oligonucleótidos antisentido y técnicas de edición génica CRISPR/Cas. Para más información sobre el grupo, consultar: www.arechavala-lab.com.

 

  • Se requiere:

Licenciatura o grado en ciencias de la salud.

Expediente académico superior a 2.5 (escala 1-4) o superior a 8 en escala 1-10.

Estar cursando/haber cursado un Máster Universitario Oficial que le garantice el acceso a un programa de Doctorado en el curso 2016/2017.

Excelentes habilidades comunicativas y organizativas.

Manejo de programas informáticos (Excel, Word, Access, etc).

Idiomas: inglés hablado y escrito (nivel muy alto).

Disponibilidad para viajar.

Capacidad para trabajar independientemente así como en equipo.

 

  • Se valora:

Conocimiento y experiencia en técnicas de Biología Molecular: extracción y purificación de DNA, RNA y proteínas, transformación y transfección, PCR.

Experiencia en cultivo celular.

Experiencia en PCR  cuantitativo.

Experiencia en manejo de muestras biológicas.

Experiencia en técnicas de histología: procesamiento de tejidos, fijación e inclusión, tinciones histológicas básicas.

Experiencia en técnicas de inmunohistoquímica e inmunofluorescencia.

Experiencia en el uso de vectores virales.

Conocimiento del manejo de programas estadísticos

Idiomas: euskera hablado y escrito.

 

Contacto: virginia.arechavalagomeza@osakidetza.eus

Lancet Neurology Policy View: Stakeholder cooperation in orphan medicine development

Finally, a paper that originanted at the EMA DMD stakeholder meeting last year was published today in Lancet Neurology. Cooperation amongst stakeholders is vital, and you can see how deeply we believe in this: this is a picture of some of the authors of this paper.

SCOPE-DMD meeting: Making outcomes work

Clinicians, researchers, regulators and patients met in London last 26th of May to discuss how to best find and validate outcome measures meaningful for patients and acceptable to regulators. It was a great opportunity to discuss the latest advances on this issue.

 

 

 

How to write a paper with many, many authors:

  1. Lock many scientists in a room
  2. Give them food and coffee and LOTS OF POST-ITS
  3. FOCUS
  4. Outline ready to go! (Now we only need to fill in the gaps… watch this space in the upcoming months).

“GIOCONDA SALUD” VISIT

 

Last month we were visited by some high school students taking part in the “Gioconda Salud” initiative. They visited several of Cruces University Hospital’s departments, including ours at BioCruces. We told them about the uses of diagnostic tissue leftovers in research and send them home with a mission… spread the word, sign informed consents and help research!

Rare disease day 2016

This year the rare disease day is extra-rare and we have team up with other researchers at BioCruces Health Research Institute to campaign for FEDER (Federación Española de Enfermedades Raras).

 

BioCruces Rare Researchers…

COST WORKSHOP BILBAO 2-3-4 Feb 2016

Recently, we hosted at BioCruces two back to back COST workshops (one on systemic delivery and the second on CNS delivery of antisense). 45 researchers from 11 different European countries (and a few extra nationalities) met up to discuss their latest results on the subject and there were plenty of opportunities for consolidated and early career scientist to network. It was a pleasure to finally greet our colleagues at home and are looking forward to a new visit.

Attendants to the first workshop, on systemic delivery of antisense oligonucleotides.

Attendants to the second workshop on antisense delivery to the CNS.

We have moved!

Over the last few weeks our group has moved to a new location within BioCruces Health Research Institute.
The new laboratories and office space are located at “Laboratorios de Investigación BioCruces 3”, where the old microbiology laboratories of the hospital used to be. After a total overhaul of the building, there are now two open plan floors of laboratory space and separated tissue culture, microscopy and flow citometry platforms. You may find us in this map or in the googlemaps link below:

https://www.google.es/maps/place/BioCruces+Health+Research+Institute/@43.283177,-2.9868697,17z/data=!3m1!4b1!4m2!3m1!1s0xd4e5085ba69e087:0x37538dbad77d7143

We are looking for a pre-doctoral candidate

We are looking for candidates to apply for funding together. A copy of the ad, in spanish, is below. If you are interested, contact us.

SE BUSCA CANDIDAT@ PREDOCTORAL EN EL GRUPO DE ENFERMEDADES NEUROMUSCULARES DEL IIS BIOCRUCES

Buscamos candidatos para optar a becas predoctorales de concurso público y realizar una tesis doctoral en el grupo de enfermedades neuromusculares del instituto de investigación sanitaria BioCruces.

El grupo de Enfermedades Neuromusculares centra su interés en la evaluación de nuevos compuestos terapéuticos para estas enfermedades, en particular la distrofia muscular de Duchenne. Actualmente está realizando proyectos para evaluar oligonucleótidos antisentido y técnicas de edición génica CRISPR/Cas. Para más información sobre el grupo, consultar: www.arechavala-lab.com.

• Se requiere:
Licenciatura o grado en ciencias de la salud.
Expediente académico superior a 2.5 (escala 1-4) o superior a 8 en escala 1-10.
Estar cursando/haber cursado un Máster Universitario Oficial que le garantice el acceso a un programa de Doctorado en el curso 2016/2017.
Excelentes habilidades comunicativas y organizativas.
Manejo de programas informáticos (Excel, Word, Access, etc).
Idiomas: inglés hablado y escrito (nivel muy alto).
Disponibilidad para viajar.
Capacidad para trabajar independientemente así como en equipo.

• Se valora:
Conocimiento y experiencia en técnicas de Biología Molecular: extracción y purificación de DNA, RNA y proteínas, transformación y transfección, PCR.
Experiencia en cultivo celular.
Experiencia en PCR cuantitativo.
Experiencia en manejo de muestras biológicas.
Experiencia en técnicas de histología: procesamiento de tejidos, fijación e inclusión, tinciones histológicas básicas.
Experiencia en técnicas de inmunohistoquímica e inmunofluorescencia.
Experiencia en el uso de vectores virales.
Conocimiento del manejo de programas estadísticos
Idiomas: euskera hablado y escrito.

Contacto: virginia.arechavalagomeza@osakidetza.eus

Spanish Translation of DMD research overview

Dr Arechavala-Gomeza has translated again into spanish the DMD research overview that is mantained in English by Dr Amenieke Aartsma-Rus.

You may find it at the TREAT-NMD webpage or here.

World Muscle Society Congress 2015

Dr Arechavala-Gomeza presented our poster “Quantifying dystrophin in cell culture: a method to accelerate preclinical assessment of DMD treatments” at the WMS meeting, held in Brighton, UK (http://wms2015.com/ 30 Sept-4 Oct).
If you could not make it to Brighton, a pdf our poster is in the poster section.

While in Brighton, preparations started for next year’s congress in Granada. See you all there!

Pictures by @_simoncallaghan

 

 

 

World Duchenne Awareness Day 7th Sept 2015

This is a very interesting short video on Duchenne Muscular Dystrophy (DMD), by www.worldduchenneawarenessday.org

Ice Bucket Challenge 2015

It has been a year since we took part in the original ice bucket challenge to ask for donations for research in ALS. As there is plenty to do, we have done it again… Enjoy!

 

World Duchenne Awareness Day 7th September 2015

Next 7th of September is Duchenne Awareness Day and today we are leaving this space to www.worldduchenneday.org:

“One in 3,500 newborn boys in the world is affected by a rare and fatal disease: Duchenne muscular dystrophy. One in 3,500 might seem a drop in the Ocean of whole human race if we merely focus our attention on statistics. Moving from data to value, the value of life, we can discover that that ´drop´ forms a great sea made up of families, researchers, volunteers, clinicians, activists. All of them work day by day to guarantee access to care, to improve research, to educate patients and families. Education can turn a problem into a solution, pain into strength. This is why World Duchenne Awareness Day 2015 is has launched the Duchenne Balloon campaign focused on supporting education and awareness programs: a worldwide informed community is the key to a better future. No matter where you live or how old you are, you can be another drop in our ocean! Join and discover World Duchenne Awareness Day: All together we can build a more inclusive world where equal rights and opportunities are guaranteed, differences celebrated and a cure for Duchenne muscular dystrophy discovered. The World Duchenne Awareness Day got the Patronage of the European Parliament!”

"Let it grow", a tissue culture athem.

Some months ago, a colleague recomended we checked out this song: a cover version of Frozen’s “Let it go” adapted to tissue culture lab work. It was  produced by high school student interns at the City of Hope in Duarte, California funded through a grant by California’s Stem Cell Agency (CIRM).

The video can be seen at California Institute for Regenerative Medicine’s youtube channel.

 

 

 

Videos of the DMD workshop hosted by the European Medicines Agency

We recently attended a stakeholder meeting hosted by the EMA and organised by COST Action BM1207 and SCOPE-DMD. It was a very interesting day and EMA has just uploaded all 16 recordings of the presentations of the day.
You may watch them at EMA’s youtube channel here or below. To select a particular video use the playlist icon at the bottom of the screen.

ASEM Catalunya Meeting June 2015

Dr Arechavala-Gomeza has been invited to participate in the symposium organised by ASEM Catalunya (Associació Catalana de Persones amb Malalties Neuromusculars) to discuss basic research in neuromuscular disorders. You may find the program here.

COST workshop in Munich. May 2015

Dr Arechavala-Gomeza joined other COST members in Munich for the latest combined management committee meeting and workshop of our COST Action on exon skipping. As usual, a lot was learned during a couple of very interesting days. Thank you to the organisers for the seamless run of the meeting and great venue.

COST exon skipping workshop in Leiden, the Netherlands 19th May 2015

COST exon skipping workshop in Leiden, the Netherlands.
Dr García-Jímenez and Dr Arechavala-Gomeza joined colleagues from several research institutions in Leiden to participate in a COST-funded workshop to discuss methods used to evaluate exon skipping and RNA stability in DMD samples. We thank our colleagues from UCL, University of Ferrara, Royal Holloway and BioMarin for the fruitful discussion and our hosts at the DMD Genetic Therapy Group at Leiden University Medical Center (LUMC) for their hospitality.

Anniversary of BENE May 2015

Anniversary of the Association of neuromuscular patients of Biscay (BENE). May 2015
We thank BENE (the local association of neuromuscular patients), for their invitation to attend the seminars organized for their 18th anniversary. It was a pleasure to participate in these celebrations.

 

Seminar at Galdakao Hospital. 11th May 2015

Seminar at Galdakao Hospital. 11th May 2015

Recently, Dr Arechavala-Gomeza was invited to give a seminar at Galdakao Hospital titled: “Terapias avanzadas: más cerca de la clínica de lo que parece. (Lecciones aprendidas de la distrofia muscular de Duchenne.)” (Advanced therapies: closer to the bedside tan you may think, lessons from Duchenne muscular dystrophy experience).
Members of Osakidetza (Basque Health Service) have access to a recording of this presentation here.

Science + Meeting, Bilbao 7-9 May 2015

A few days ago we joined our friends at the Spanish Scientist associations in Germany (CERFA) and the UK (SRUK) and the University of the Basque Country in a meeting that presented, in a very welcoming setting, the different professional options in research careers: from academia to industry, science communication and science journalism, journal editing, as well as funding opportunities in different countries, and science policy. All seminars were delivered by very experienced professionals and policy makers. The general consensus amongst those of us that had the privilege to attend was: “I wish this had been organised while I was starting my PhD”.
We are looking forward to Science Plus 2016.

 

 

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