Common therapeutic advances for Duchenne muscular dystrophy (DMD).

Background and purpose: Duchenne muscular dystrophy (DMD), a lethal X-linked recessive muscle dystrophy, is resulted in by different mutations including mostly frame-shifting gross deletions and duplications and rarely point mutations in DMD gene. Increasing weakness, progressive loss of skeletal muscle mass, and later-onset cardiomyopathy are serious clinical symptoms which ultimately lead to cardiac and respiratory failure, and premature death in DMD patients by age of 30. DMD is a prevalent genetic disorder and considers as an interesting target for gene therapy approaches. Massive gene size and existence of enormous number of muscle tissues are terrific hindrance against DMD treatments, nevertheless enormous efforts have been executed in the fields of gene replacement therapy, gene editing strategies, cell-based treatments, and small drug medications. Hot spot exons skipping and suppression of premature stop codons are the most interesting treatments for restoring functional DMD product, dystrophin protein. Clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein (Cas) systems are the most interesting genome editing platforms that are able to restore open reading frame of DMD gene. CRISPR-Cas9 and CRISPR-Cpf1 are two main genome editing sub-types that successfully used in mdx mice.Conclusions: This review aims to present recent progresses and future prospects over three main DMD therapeutic subgroups including gene therapy, cell therapy, and pharmacological therapy.

Spinal Muscular Atrophy and Common Therapeutic Advances.

BACKGROUND: Spinal muscular atrophy (SMA) is an autosomal recessive destructive motor neuron disease which is characterized primarily by the degeneration of α-motor neurons in the ventral gray horn of the spinal cord. It mainly affects children and represents the most common reason of inherited infant mortality. MATERIAL AND METHODS: We provide an overview of the recent therapeutic strategies for the treatment of SMA together with available and developing therapeutic strategies. For this purpose, Google Scholar and PubMed databases were searched for literature on SMA, therapy and treatment. Titles were reviewed and 96 were selected and assessed in this paper. RESULT: Over the last two decades, different therapeutic strategies have been proposed for SMA. Some methods are in the pre-clinical, others the clinical phase. CONCLUSION: By emergence of the new approaches, especially in gene therapy, effective treatment in the close future is probable.

Recent advances in CRISPR-Cas systems for colorectal cancer research and therapeutics.

INTRODUCTION: Colon cancer, ranked as the fourth leading global cause of cancer death, exhibits a complex progression marked by genetic variations. Over the past decade, the utilization of diverse CRISPR systems has propelled accelerated research into colorectal cancer (CRC) treatment. AREAS COVERED: CRISPR/Cas9, a key player in this research, identifies new oncogenes, tumor suppressor genes (TSGs), and drug-resistance genes. Additionally, it facilitates the construction of experimental models, conducts genome-wide library screening, and develops new therapeutic targets, especially for targeted knockout in vivo or molecular targeted drug delivery, contributing to personalized treatments and significantly enhancing the care of colon cancer patients. In this review, we provide insights into the mechanism of the CRISPR/Cas9 system, offering a comprehensive exploration of its applications in CRC, spanning screening, modeling, gene functions, diagnosis, and gene therapy. While acknowledging its transformative potential, the article  highlights the challenges and limitations of CRISPR systems. EXPERT OPINION: The application of CRISPR/Cas9 in CRC research provides a promising avenue for personalized treatments. Its potential for identifying key genes and enabling experimental models and genome-wide screening enhances patient care. This review underscores the significance of CRISPR-Cas9 gene editing technology across basic research, diagnosis, and the treatment landscape of colon cancer.

Pathophysiology and diagnosis of diabetic retinopathy: a narrative review.

Diabetes is an endocrine disorder which is known by abnormal high blood glucose levels. There are two main categories of diabetes: type I (10%-15%) and type II (85%-90%). Although type II is more common, type I is the most common form in children. Diabetic retinopathy (DR), which remains the foremost cause of losing vision in working-age populations, can be considered as the main complication of diabetes mellitus. So choosing the best method for diagnosing, tracking, and treating the DR is vital to enhance the quality of life and decrease the medical expenses. Each method for diagnosing DR has some advantages and the best way must be selected according to the points that we need to find. For writing this manuscript, we made a list of relevant keywords including diabetes, DR, pathophysiology, ultrawide field imaging, fluorescein angiography, optical coherence tomography, and optical coherence tomography-angiography, and then we started searching for studies in PubMed, Scopus, and Web of Science databases. This review article covers the pathophysiology of DR and medical imaging techniques to monitor DR. First, we introduce DR and its pathophysiology and then we present the medical imaging techniques to monitor it.

An Overview of the CRISPR-Based Genomic- and Epigenome-Editing System: Function, Applications, and Challenges.

Developing a new strategy for an efficient targeted genome editing has always been a great perspective in biology. Although different approaches have been suggested in the last three decades, each one is confronting with limitations. CRISPR-Cas complex is a bacterial-derived system which made a breakthrough in the area of genome editing. This paper presents a brief history of CRISPR genome editing and discusses thoroughly how it works in bacteria and mammalians. At the end, some applications and challenges of this growing research area are also reviewed. In addition to moving the boundaries of genetics, CRISPR-Cas can also provide the ground for fundamental advances in other fields of biological sciences.