Saeed Anwar

Genetic therapeutics researcher | Rare disease advocate | Sci-activist | Bengali SciFi writer | Overcaffeinated | Foodie | First-gen | 🇧🇩
♡︎/⟳ ≠ endorsement

[Saeed Anwar @SaeedAnuj]

DMD-Null mice (i.e., lacking all dystrophin isoforms) exhibit severe muscle weakness and poor regeneration. This proves that shorter dystrophin isoforms do aid regeneration to some extent.
https://t.co/fPU68r9qPy

[Saeed Anwar @SaeedAnuj]

Bottlebrush polymer that protects muscle membranes. Interesting development. https://t.co/9Pl4hNca23.

[JACC Journals @JACCJournals]

Micro-dystrophin gene therapy holds promise for #DMD. In this #JACCBTS article, dystrophin #GeneTherapy in 🐀 model 📈 survival but also ⬆️cardiac inflammation. Understanding immune responses to these therapies is 🔑 for developing safer Txs. https://t.co/C1R3nZIMCI #JACCBTS

[Saeed Anwar @SaeedAnuj]

@ToshifumiYokota Thank you so much, Dr. Yokota! I am truly grateful for the mentorship and resources you have provided, and for the scientific trust and freedom you afforded me throughout my grad school.
I hope to use the knowledge I gained from this experience to benefit others.

[Saeed Anwar @SaeedAnuj]

@ewglcnt @MolTherapy Thanks for your comment. Our study shows promising functional recovery in vitro, but we still need in vivo validation. While we've done some in silico evaluations, our next step is a deeper in vivo study to assess long-term efficacy and safety.

[Saeed Anwar @SaeedAnuj]

@sciqst @MolTherapy Thank you for your insightful comment! ASOs indeed have great potential, but scalability is challenging due to cost and delivery. We're actively working on advanced delivery systems to improve efficacy, safety, and accessibility.

[Arc Institute @arcinstitute]

Today, we're launching the Arc Virtual Cell Atlas, a growing resource for computation-ready single-cell measurements. As the initial contributions, @vevo_ai has open sourced Tahoe-100M, the world's largest single-cell dataset, mapping 60,000 drug-cell interactions, and we’re announcing scBaseCamp, the first RNA sequencing data repository curated using AI agents. Combined, the release includes data from over 300 million cells.

https://t.co/OrjRT6YwWS

[ASGCT @ASGCTherapy]

Congratulations to our #ASGCTBreakthroughs24 Outstanding Poster Award winners!

- Saeed Anwar (@UAlberta)

- Pranali Mistry, (@nationwidekids)

You can read these abstracts and the rest of the collection on our website. https://t.co/HUWnz2YcZI

[Saeed Anwar @SaeedAnuj]

@ASGCTherapy @UAlberta @nationwidekids Thank you, @ASGCTherapy! It was an absolute privilege to share our work at #ASGCTBreakthroughs24. It was a fantastic opportunity for knowledge sharing and networking. Congrats to the fellow award winner! Thanks, @MDAorg and everyone else who contributed to this amazing event.

[Prof. Toshi Yokota PhD FCAHS @MD_Canada Res Chair @ToshifumiYokota]

Thrilled to share the incredible achievement of our PhD student, Saeed, who has won the Outstanding Poster Award! 🏆His work on enhancing #AntisenseOligo efficacy with small molecule activity enhancers is groundbreaking. Congrats, Saeed!🎉@ASGCTherapy @WCHRIUofA @UAlberta_FoMD

[Saeed Anwar @SaeedAnuj]

@ToshifumiYokota @ASGCTherapy @WCHRIUofA @UAlberta_FoMD Thank you, Dr. Yokota! I am honored to receive this award and sincerely appreciate your mentorship and support. I look forward to our ongoing work to enhance ASO efficacy using the many fantastic methods we are developing in the lab!

[Saeed Anwar @SaeedAnuj]

Excited to attend the #ASGCTBreakthroughs24 conference by @ASGCTherapy and @MDAorg in Chicago! If you are around, please stop by my poster on improving antisense oligonucleotide efficacy with small-molecule enhancers.
📍 Poster #44
🗓️ Tuesday, Nov 17th, 5:45 PM
See you there!

[The Nobel Prize @NobelPrize]

BREAKING NEWS
The 2024 #NobelPrize in Physiology or Medicine has been awarded to Victor Ambros and Gary Ruvkun for the discovery of microRNA and its role in post-transcriptional gene regulation.

[Saeed Anwar @SaeedAnuj]

This is true for more than just dysferlinopathies!
People with the same genetic disease often miss clinical trials because their symptoms/conditions have different names. A unified system based on genetic causes, NOT symptoms, would help more patients access trials & treatments.

[Katsuya Yagyu @Yagyu2p13]

Clinical trials for LGMD R2/LGMD 2B are currently underway, but patients with other Dysferlin gene mutations cannot participate. Therefore, it has attracted attention from LGMD R2/LGMD 2B patients, but no response from MMD/MM, DMAT, or DACM patients.

The reason is that because the disease names are categorized based on the phenotype, many people are unaware of the following:🥲
1. I don't know that it's a muscle disease that develops with the same causative gene
2. Not knowing that they can be treated together
3. There are cases in which even related Brothers and sisters have different disease names
*If the medical institution is different

Pharmaceutical companies will never conduct clinical trials on MMD/MM after LGMD R2/LGMD 2B. The reason for this is that it takes a lot of effort, time, and research expenses. Also, not everyone can take part in a clinical trial.

If things continue as they are, I think it is possible that some countries will not be able to recruit participants and clinical trials will not be able to be conducted, or that the trials will end prematurely. The reason for this is that although it is a rare disease, muscle diseases with the same causative gene are classified by phenotype.

I think this is somewhat of a tragic situation.😔💔
I hope that people with the same disease will seriously consider this issue.🌎🌍🌏

#MiyoshiMuscularDystrophy #MiyoshiMyopathy
#LGMDR2 #LGMD2B #DMAT #DACM
#Dysferlin
#Dysferlinopathy
#MuscularDystrophy
#WorldMuscleSociety

[Saeed Anwar @SaeedAnuj]

It's been 4 days since I last spoke to my family back home. Telecommunication is completely shut down. A TV centre near my place was torched. The attackers keep on targeting innocent and unarmed #students all over #Bangladesh. God knows what's happening there.
So suffocating!

[Saeed Anwar @SaeedAnuj]

Feeling helpless and ashamed.
Nearly 70 innocent souls lost, including minors, and hundreds injured.
Media & communication are heavily restricted, making it hard for the truth to come out. Can't contact my family and friends.
#Bangladesh #EndViolence

[philly cheesesteak @awwtyzm]

this live network data shows #Bangladesh is in the midst of a near total nationwide internet blackout.

clearly an early warning of an overnight genocide against protesters, to cripple their communication for aid and info.

#SaveBangaldeshiStudents

[Ha San @HaSan6484]

Right now there is no electricity and internet connection in Bangladesh, now there is a total blackout please save Bangladesh,Today, more than 50 students have been shot dead by the government and police and more than 10,000 students have been injured in the student movement.

[Saeed Anwar @SaeedAnuj]

@AMVoronova @NatureComms @vizgen_inc @UAlberta_FoMD @KBGFdn @WCHRIUofA Congratulations to the entire team!

[Saeed Anwar @SaeedAnuj]

@AMVoronova @adrianne_neuro @KarimiLab @jrplemel Congratulations to Dr. Watson, and kudos to you, Dr. Voronova, for the excellent mentorship you have provided!